Genetic Disorders

An FDA approved indication to reduce the frequency of vaso-occlusive crises (VOCs) in adult members and in pediatric members 16 years of age and older with sickle cell disease (SCD); AND
Member must have a history of VOCs; AND
Adakveo® must be prescribed by, or in consultation with, a hematologist or a specialist with expertise in treatment of SCD (or an advanced care practitioner with a supervising physician who is a hematologist or specialist with expertise in treating SCD); AND
Prescriber must verify Adakveo® will be administered by a trained health care provider. The prior authorization request must indicate how Adakveo® will be administered; and
- Adakveo® must be shipped via cold chain supply to the facility where the member is scheduled to receive treatment; OR
- Adakveo® must be shipped via cold chain supply to the member’s home and administered by a home health provider, and the member or member’s caregiver must be trained on the proper storage of Adakveo®; AND
A recent (within the last 3 months) weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; AND
Approval quantities will be dependent on the member’s weight and will include loading doses at week 0 and 2, then subsequent doses every 4 weeks in accordance with package labeling; AND
Initial approvals will be for the duration of 3 months. Subsequent approvals will be for 1 year if the prescriber documents the member is responding well to treatment.

Prolastin®-C Liquid [Alpha1-Proteinase Inhibitor (Human)] Approval Criteria:

An FDA approved indication for augmentation and maintenance therapy of patients 18 years of age or older with severe hereditary deficiency of alpha1-antitrypsin (AAT) with clinical evidence of emphysema; AND
Diagnosis confirmed by all of the following:
- Genetic confirmation of PiZZ, PiZ(null) or Pi(null, null) phenotype alpha1-antitrypsin deficiency (AATD) or other alleles determined to increase risk of AATD; AND
- Serum levels of AAT less than 11µmol/L; AND
- Documented emphysema with airflow obstruction; AND
Prescriber must document that member’s forced expiratory volume in one second (FEV1) is less than or equal to 65% predicted; AND
Must be prescribed by a pulmonary disease specialist or advanced care practitioner specializing in pulmonary disease; AND
The prescriber must verify the member is a non-smoker; AND
The prescriber must verify the member does not have antibodies to IgA; AND
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling.

Aralast NP™,Glassia®, and Zemaira® (Alpha1-Proteinase Inhibitor [Human]) Approval Criteria:

An FDA approved indication for augmentation and maintenance therapy of patients 18 years of age or older with severe hereditary deficiency of alpha1-antitrypsin (AAT) with clinical evidence of emphysema; AND
Diagnosis confirmed by all of the following:
Genetic confirmation of PiZZ, PiZ(null), or Pi(null, null) phenotype alpha1-antitrypsin deficiency (AATD) or other alleles determined to increase risk of AATD; AND
Serum levels of AAT less than 11µmol/L; AND
Documented emphysema with airflow obstruction; AND
Prescriber must document that member’s forced expiratory volume in one second (FEV1) is less than or equal to 65% predicted; AND
Must be prescribed by a pulmonary disease specialist or advanced care practitioner specializing in pulmonary disease; AND
The prescriber must verify the member is a non-smoker; AND
The prescriber must verify the member does not have antibodies to IgA; AND
A patient-specific, clinically significant reason why the member cannot use Prolastin®-C; AND
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling.

Prior Authorization form

Amvuttra™ (Vutrisiran) and Onpattro™ (Patisiran) Approval Criteria:

An FDA approved indication for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis; AND
Diagnosis confirmed by the following:
- Tissue (fat pad) biopsy confirming amyloid deposits; AND
- Genetic confirmation of transthyretin (TTR) gene mutation (e.g., Val30Met); AND
Prescriber must verify member is currently experiencing signs and symptoms of polyneuropathy and other causes of polyneuropathy have been ruled out; AND
Onpattro™ must be prescribed by or in consultation with a cardiologist, geneticist, or neurologist or an advanced care practitioner with a supervising physician who is a cardiologist, geneticist, or neurologist; AND
Prescriber must confirm the member will take the recommended daily allowance of vitamin A; AND
Prescriber must confirm the member does not have severe renal impairment, end-stage renal disease, and/or moderate or severe hepatic impairment; AND
Prescriber must confirm the member has not undergone a liver transplant; AND

For Onpattro^®, prescriber must confirm that member will be pre-medicated with intravenous (IV) corticosteroid, oral acetaminophen, IV histamine-1 (H1) antagonist, and IV histamine-2 (H2) antagonist 60 minutes prior to Onpattro™ administration to reduce the risk of infusion-related reactions; AND
Amvuttra™ will not be approved for concomitant use with Onpattro^® (patisiran), Tegsedi^® (inotersen), Vyndamax^® (tafamidis), or Vyndaqel^® (tafamidis meglumine); AND
Authorization for Amvuttra™ will also require a patient-specific, clinically significant reason why the member cannot use Onpattro^®; AND
Onpattro™ will not be approved for concomitant use with Amvuttra™ (vutrisiran), Tegsedi™,Vyndaqel® (tafamidis meglumine), or Vyndamax™ (tafamidis); AND
For Onpattro^®, member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; AND
For Amvuttra™, a quantity limit of 0.5mL per 90 days will apply; AND
Onpattro™ approvals will be for the duration of 1 year. Reauthorization may be granted if the prescriber documents the member is responding well to treatment and member has not undergone a liver transplant.

Prior Authorization form

Bylvay™ (Odevixibat) Approval Criteria:

An FDA approved indication for the treatment of pruritus in members with progressive familial intrahepatic cholestasis (PFIC); and
- Diagnosis must be confirmed by genetic testing identifying mutations in the ATP8B1, ABCB11, or ABCB4 genes; and
Member must be 3 months of age or older; and
Bylvay™ must be prescribed by a gastroenterologist, hepatologist, geneticist, or other specialist with expertise in the treatment of PFIC (or an advanced care practitioner with a supervising physician who is a gastroenterologist, hepatologist, geneticist, or other specialist with expertise in the treatment of PFIC); and
Prescriber must verify member has a history of significant pruritus that is unresponsive to treatment with ursodeoxycholic acid (UDCA) and at least 2 of the following medications, unless contraindicated:
- Cholestyramine; or
- Rifampin; or
- Sertraline; or
- Naltrexone; and
Member must have elevated serum bile acid concentration ≥100micromol/L at baseline; and
Prescriber must verify member does not have known pathologic variants of the ABCB11 gene predicting a non-functional or absent bile salt export pump protein (BSEP-3); and
Members with a history of liver transplantation will generally not be approved for Bylvay™; and
Prescriber must verify surgical intervention (e.g., biliary diversion, liver transplantation) is not currently clinically appropriate for the member; and
Prescriber must agree to monitor alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin, direct bilirubin, and international normalized ratio (INR) at baseline and during treatment with Bylvay™; and
Member’s current weight (taken within the past 3 weeks) must be provided on initial and subsequent prior authorization requests in order to authorize the appropriate amount of drug required according to package labeling; and
Initial approvals will be for 40mcg/kg/day for a duration of 3 months. After 3 months of treatment, further approval may be granted at the 40mcg/kg/day dose if the prescriber documents the member is responding well to treatment and surgical intervention is still not clinically appropriate; or
Dose increases to 80mcg/kg/day (for 3 months) and 120mcg/kg/day (for 3 months) may be approved if there is no improvement in pruritus after 3 months of treatment with the lower dose(s). Further approval may be granted if the prescriber documents the member is responding well to treatment at the current dose and surgical intervention is still not clinically appropriate; and
If there is no improvement in pruritus after 3 months of treatment with the maximum 120mcg/kg/day dose, further approval of Bylvay™ will not be granted.

Carbaglu® (Carglumic Acid) Approval Criteria:

An FDA approved diagnosis of 1 of the following:
- Adjunctive therapy to the standard of care for the treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency; OR
- Maintenance therapy for the treatment of chronic hyperammonemia due to NAGS deficiency; OR
- Adjunctive therapy to the standard of care for the treatment of acute hyperammonemia due to propionic acidemia (PA) or methylmalonic acidemia (MMA); AND
Carbaglu® must be prescribed by, or in consultation with, a geneticist; AND
Carbaglu is brand preferred; use of generic carglumic acid will require a patient-specific, clinically significant reason why the member cannot use the brand formulation; AND
For a diagnosis of hyperammonemia due to NAGS deficiency:
- Documentation of active management with a low protein diet; AND
- Initial approvals will be for the duration of 1 year. After that time, reauthorization will require the prescriber to verify the member is responding well to therapy.
For a diagnosis of acute hyperammonemia due to PA or MMA:
- Documentation the member’s plasma ammonia level is ≥50micromol/L and is not due to liver failure; AND
- Prescriber must confirm Carbaglu^® is being used concurrently with other ammonia-lowering therapies [e.g., intravenous (IV) glucose, insulin, L-carnitine, protein restriction, dialysis]; AND
- Number of days Carbaglu^® was received while hospitalized must be provided; AND
- Approvals will be for no longer than 7 days total (including treatment days while hospitalized) as there is currently no evidence to support the use of Carbaglu^® for acute hyperammonemia due to PA or MMA beyond 7 days.

Prior Authorization form

Crysvita® (Burosumab-twza) Approval Criteria [X-Linked Hypophosphatemia (XLH) Diagnosis]:

An FDA approved indication for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older. Diagnosis of XLH must be confirmed by one of the following:
- Genetic testing; OR
- Elevated serum fibroblast growth factor 23 (FGF23) level; AND
Member’s serum phosphorus level must be below the normal range for member age; AND
Member must not have any contraindications to taking Crysvita® including the following:
- Concomitant use with oral phosphate and active vitamin D analogs; AND
- Serum phosphorus within or above the normal range for member age; AND
- Severe renal impairment or end-stage renal disease; AND
Crysvita® must be administered by a health care professional. Approvals will not be granted for self-administration. Prior authorization requests must indicate how Crysvita® will be administered; AND
- Crysvita® must be shipped via cold chain supply to the facility where the member is scheduled to receive treatment; AND
- Crysvita® must be shipped via cold chain supply to the member’s home and administered by a home health care provider and the member’s caregiver must be trained on the proper storage of Crysvita®; AND
Member must have clinical signs and symptoms of XLH (symptoms beyond hypophosphatemia alone); AND
Every two week dosing will not be approved for members 18 years of age or older; AND
The prescriber must agree to assess serum phosphorus levels on a monthly basis for the first 3 months of treatment, and thereafter as appropriate; AND
Crysvita® must be prescribed by a nephrologist, endocrinologist, or specialist with expertise in the treatment of XLH (or be an advanced care practitioner with a supervising physician who is a nephrologist, endocrinologist, or specialist with expertise in the treatment of XLH); AND
Initial authorizations will be for the duration of 6 months, at which time the prescriber must verify the member is responding to the medication as demonstrated by serum phosphorus levels within the normal range for member age or clinically significant improvement in bone-related symptoms; AND
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling.

Crysvita^® (Burosumab-twza) Approval Criteria [Tumor-Induced Osteomalacia (TIO) Diagnosis]:

An FDA approved diagnosis for the treatment of fibroblast growth factor 23 (FGF-23)-related hypophosphatemia in TIO associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in members 2 years of age and older; and
Member’s diagnosis must be confirmed by elevated serum FGF23 level that was not amendable to cure by surgical excision of the underlying tumor/lesion; and
Member’s serum phosphorus level must be below the normal range for member age; and
Member must not have any contraindications to taking Crysvita^® including the following:
- Concomitant use with oral phosphate and active vitamin D analogs; and
- Serum phosphorus within or above the normal range for member age; and
- Severe renal impairment or end-stage renal disease; and
Crysvita^® must be administered by a health care professional. Approvals will not be granted for self-administration. Prior authorization requests must indicate how Crysvita^® will be administered; and
- Crysvita^® must be shipped via cold chain supply to the facility where the member is scheduled to receive treatment; or
- Crysvita^® must be shipped via cold chain supply to the member’s home and administered by a home health care provider if the member’s caregiver has been trained on the proper storage of Crysvita^®; and
The prescriber must agree to assess serum phosphorus levels on a monthly basis for the first 3 months of treatment and thereafter as appropriate and follow the Crysvita^® Prescribing Information for dose adjustments; and
The prescriber must agree to monitor 25-hydroxy vitamin D levels; and
Crysvita^® must be prescribed by an endocrinologist or specialist with expertise in the treatment of TIO (or an advanced care practitioner with a supervising physician who is an endocrinologist or specialist with expertise in treating TIO); and
The member’s recent weight (within the last 3 months) must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; and
Initial authorizations will be for the duration of 6 months, at which time the prescriber must verify the member is responding to the medication as demonstrated by serum phosphorus levels within the normal range for member age or clinically significant improvement in bone-related symptoms; and
Early refill requests for dose changes more frequently than every 4 weeks will not be approved; and
The maximum approvable dosing regimen is 180mg every 2 weeks; and
A quantity limit of 12 single-dose vials per month will apply.

Prior Authorization form

Cuvrior™ (Trientine Tetrahydrochloride) Approval Criteria:

An FDA approved diagnosis of Wilson’s disease; and
- Diagnosis must be confirmed by a Leipzig score ≥4; and
Member must be 18 years of age or older; and
Cuvrior must be prescribed by, or in consultation with, a gastroenterologist, hepatologist, or other specialist with expertise in the treatment of Wilson’s disease (or an advanced care practitioner with a supervising physician who is gastroenterologist, hepatologist, or other specialist with expertise in the treatment of Wilson’s disease); and
Member must be clinically stable, de-coppered, and tolerant to penicillamine as indicated by 1 of the following:
- Serum non-ceruloplasmin copper (NCC) level 25-150mcg/L; or
- Urinary copper excretion (UCE) level 200-500mcg/24 hours; and
Prescriber must verify the member will discontinue therapy with penicillamine or other copper chelating agents prior to starting therapy with Cuvrior™; and
A patient-specific, clinically significant reason why the member cannot use penicillamine, generic trientine hydrochloride, and Galzin^® (zinc acetate), which are available without a prior authorization, must be provided; and
A quantity limit of 288 tablets per 28 days will apply.

Daybue™ (Trofinetide) Approval Criteria:

Diagnosis of typical Rett syndrome confirmed by all of the following:
- Prescriber must verify all clinical diagnostic criteria are met supporting a diagnosis of typical Rett syndrome including:
  - A period of regression followed by recovery or stabilization; and
  - Partial or complete loss of acquired purposeful hand skills; and
  - Partial or complete loss of acquired spoken language; and
  - Gait abnormalities (impaired/dyspraxic or absence of ability); and
  - Stereotypic hand movements (e.g., hand wringing/squeezing, clapping/tapping, mouthing, washing/rubbing automatisms); and
  - Lack of brain injury secondary to trauma (peri- or postnatally), neurometabolic disease, or severe infection causing neurological problems; and
  - Lack of grossly abnormal psychomotor development in the first 6 months of life; and
- Genetic testing documenting a disease-causing mutation in the MECP2 gene (results of genetic testing must be submitted); and
Member must be 2 years of age or older; and
Daybue™ must be prescribed by a geneticist, neurologist, or other specialist with expertise in the treatment of Rett syndrome; and
Prescriber must agree to counsel members and caregivers on the risks of diarrhea and weight loss associated with Daybue™ and agree to monitor appropriately for these adverse effects; and
Prescriber must agree to counsel members and caregivers on proper storage and administration of Daybue™, including the use of a calibrated device for measuring each dose; and
Prescriber must verify the member does not have moderate or severe renal impairment; and
Member’s current weight (kg) taken within the past 3 weeks must be provided on initial and subsequent prior authorization requests to ensure accurate weight-based dosing according to package labeling; and
Initial approvals will be for a duration of 3 months. After 3 months of treatment, further approval may be granted if the prescriber documents the member is responding well to treatment. Subsequent approvals will be for a duration of 1 year; and
A quantity limit of 3,600mL per 30 days will apply.

Dojolvi^® (Triheptanoin) Approval Criteria:

An FDA approved diagnosis of molecularly confirmed long-chain fatty acid oxidation disorder (LC-FAOD); and
Molecular testing confirms 1 of the following types of LC-FAOD:
- Carnitine-acylcarnitine translocase (CACT) deficiency; or
- Carnitine palmitoyltransferase I (CPT I) deficiency; or
- Carnitine palmitoyltransferase II (CPT II) deficiency; or
- Long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency; or
- Trifunctional protein (TFP) deficiency; or
- Very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency; and
Prescriber must verify member has a history of at least 1 significant or recurrent manifestation of LC-FAOD (e.g., cardiomyopathy, rhabdomyolysis, hypoglycemia); and
Member must have tried and failed dietary management with an alternate medium chain triglyceride (MCT) product (e.g., MCT oil) or a patient-specific, clinically significant reason why dietary management with an alternate MCT product is not appropriate for the member must be provided; and
Dojolvi^® will not be approved for concomitant use with another MCT product (other MCT products must be discontinued prior to the first dose of Dojolvi^®); and
Member must not be taking a pancreatic lipase inhibitor concomitantly with Dojolvi^®; and
Prescriber must verify the member does not have pancreatic insufficiency; and
Prescriber must verify that member or member’s caregiver has been counseled on the proper storage, preparation, and administration of Dojolvi^®, including specific considerations for use in a feeding tube, if applicable; and
Dojolvi^® must be prescribed by a geneticist or other specialist with expertise in the treatment of LC-FAOD; and
Prescriber must verify the member is under the care of a clinical specialist knowledgeable in appropriate disease-related dietary management based on member’s specific LC-FAOD and current nutritional recommendations; and
The member’s daily caloric intake (DCI) must be provided (in kcal) on the prior authorization request to verify appropriate dosing based on package labeling; and
Initial approvals will be for the duration of 3 months. After 3 months of treatment, compliance will be required, and the prescriber must verify the member has had a positive response to and is tolerating treatment with Dojolvi^®. Additionally, for members who switched from another MCT product due to adverse effects, the prescriber must verify the member has experienced fewer adverse effects with Dojolvi^®; and
Quantity limits according to package labeling will apply, with the maximum approvable dosing regimen based on a target daily dosage of Dojolvi^® up to 35% of the member’s total DCI.

Amondys 45™ (Casimersen), Exondys 51^® (Eteplirsen), Viltepso^® (Viltolarsen), and Vyondys 53™ (Golodirsen) Approval Criteria:

An FDA approved diagnosis of Duchenne muscular dystrophy (DMD); and
Member must have a confirmed mutation of the DMD gene that is amenable to exon skipping for the requested medication (results of genetic testing must be submitted); and
Member must not have previously received Elevidys (delandistrogene moxeparvovec-rokl); and
Must be prescribed by a neurologist or specialist with expertise in the treatment of DMD (or an advanced care practitioner with a supervising physician who is a neurologist or specialist with expertise in the treatment of DMD); and
Prescriber must verify the member’s renal function will be appropriately assessed prior to initiation of therapy and monitored during treatment; and
Member must be on a stable dose of a corticosteroid (at least 3 months in duration) or a patient-specific, clinically significant reason why corticosteroids are not appropriate for the member must be provided; and
A baseline assessment must be provided using at least 1 of the following exams as functionally appropriate:
- 6-minute walk test (6MWT); or
- Forced vital capacity percent predicted (FVCpp); and
The requested exon-skipping therapy will not be approved for concurrent use with any other exon-skipping therapies for DMD; and
Initial authorizations will be for the duration of 6 months, at which time the prescriber must verify the member is responding to the medication as demonstrated by clinically significant improvement or maintenance of function from pretreatment baseline status using the same exam as performed at baseline assessment; and
Subsequent approvals will be for the duration of 1 year. For yearly approvals, the prescriber must verify the member is responding to the medication as demonstrated by clinically significant improvement or maintenance of function from pretreatment baseline status using the same exam as performed at baseline assessment; and
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling.

Elevidys (Delandistrogene Moxeparvovec-rokl) Approval Criteria:

An FDA approved diagnosis of Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene (results of genetic testing must be submitted); and
Member must be 4 years through 5 years of age; and
Prescriber must attest the member is ambulatory and the results of 1 of the following tests must be submitted:
- North Star Ambulatory Assessment (NSAA); or
- 6-minute walk test (6MWT); or
- 10-meter walk test (10mWT); or
- Ascend 4 Steps; or
- Time to Rise (TTR); or
- 100-meter timed test; and
Elevidys must be prescribed by a neurologist or specialist with expertise in the treatment of DMD (or an advanced care practitioner with a supervising physician who is a neurologist or specialist with expertise in the treatment of DMD); and
Member’s baseline anti-AAVrh74 total binding antibody titers must be <1:400; and
Member must not have any deletion in exon 8 and/or exon 9 in the DMD gene; and
If the member has a deletion in the DMD gene in exon 1 to 17 and/or exons 59 to 71, the prescriber must verify the member will be monitored for a severe immune-mediated myositis reaction; and
Member must not have any active infections and if the member does have an active infection, the prescriber must verify Elevidys infusion will be postponed until infection has resolved; and
Prescriber must verify the member will initiate a corticosteroid regimen 1 day prior to the infusion of Elevidys and continue for a minimum of 60 days to reduce the risk of an immune response as specified in the package labeling; and
Prescriber must verify liver function tests (LFTs) (e.g., GGT, total bilirubin) will be performed prior to Elevidys administration and will be monitored weekly for the first 3 months following Elevidys infusion then as clinically indicated; and
Prescriber must verify troponin-I will be monitored before the Elevidys infusion and weekly for the first month following infusion then as clinically indicated; and
Prescriber must verify that platelet counts will be monitored before the Elevidys infusion and weekly for the first 2 weeks following infusion then as clinically indicated; and
Member will not be approved for concomitant treatment with exon skipping therapy (e.g., Amondys 45, Exondys 51, Viltepso^®, Vyondys 53) following Elevidys infusion (current authorizations for exon skipping therapy will be discontinued upon Elevidys approval); and
Member’s current weight (kg) taken within the past 3 weeks must be provided on the request to ensure accurate weight-based dosing according to package labeling; and
Approvals will be for 1 dose per member per lifetime.

deflazacort (Emflaza®) Approval Criteria:

An FDA approved diagnosis of Duchenne muscular dystrophy (DMD); AND
Member must be 2 years of age or older; AND
Emflaza® must be prescribed by or in consultation with a prescriber who specializes in the treatment of DMD; AND
A minimum of a six-month trial of prednisone that resulted in inadequate effects or intolerable adverse effects that are not expected to occur with Emflaza®; AND
A patient-specific, clinically significant reason why the member cannot use prednisone even when the tablets are crushed must be provided; AND
Patients already established on deflazacort via the ACCESS DMD Program must also document a patient-specific, clinically significant reason why the member cannot use prednisone even when the tablets are crushed; AND
For Emflaza® suspension, a patient-specific, clinically significant reason why the member cannot use the tablet formulation in the place of oral suspension even when the tablets are crushed must be provided; AND
Verification from the prescriber the member has had baseline eye examination; AND
For continued authorization, the member’s recent weight must be provided in order to authorize the appropriate amount of drug required according to package labeling, and the member must have had a repeat eye exam with results that are acceptable to the prescriber; AND
For the tablets, a quantity limit of 30 tablets per 30 days will apply and for the suspension, a quantity limit of 39mL (3 bottles) per 30 days will apply. Quantity limit requests will be based on the member’s recent weight taken within the last 30 days.

Prior Authorization form

Approval Criteria:

An FDA approved diagnosis of sickle cell disease; AND
Member must be at least 5 years of age or older; AND
A trial of hydroxyurea or documentation why hydroxyurea is not appropriate for the member; AND
Endari™ must be prescribed by, or in consultation with, a hematologist or a specialist with expertise in treatment of sickle cell disease (or in consultation with an advanced care practitioner with a supervising physician who is a hematologist or specialist with expertise in treating sickle cell disease); AND
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling.
Initial approvals will be for a duration of six months. Reauthorization may be granted if the prescriber documents the member is responding well to treatment.

Prior Authorization form

Enjaymo™ (Sutimlimab-jome) Approval Criteria:

An FDA approved diagnosis of primary cold agglutin disease confirmed by the following:
- Chronic hemolysis; AND
- Positive direct antiglobulin (Coombs) test for C3d; AND
- Cold agglutin titer of ≥64 at 4° Celsius; AND
Member must have 1 or more symptoms associated with cold agglutinin disease (i.e., symptomatic anemia, acrocyanosis, Raynaud’s phenomenon, hemoglobinuria, a major adverse vascular event); AND
Member has a hemoglobin (Hgb) level ≤10g/dL; AND
Member has a bilirubin level above the normal reference range; AND
Enjaymo™ must be prescribed by a hematologist (or an advanced care practitioner with a supervising physician who is a hematologist); AND
Member has not received rituximab within 3 months of initiation and will not be using rituximab concomitantly with Enjaymo™; AND
Prescriber must verify the member has been vaccinated against encapsulated bacteria (e.g., Neisseria meningitides, Streptococcus pneumoniae, Haemophilus influenzae) at least 2 weeks prior to initiation of treatment; AND
Enjaymo™ must be administered in a health care setting by a health care provider prepared to manage anaphylaxis; AND
Prescriber must agree to monitor the member for at least 2 hours following the initial infusion for signs or symptoms of an infusion and/or hypersensitivity reaction and for 1 hour following completion of subsequent infusions; AND
Prescriber must verify the member has no chronic systemic infections [e.g., hepatitis B, hepatitis C, human immunodeficiency virus (HIV)]; AND
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; AND
Initial approvals will be for 6 months. Reauthorization may be granted if the prescriber documents the member is responding well to therapy, as confirmed by at least 1 of the following:
- Member has had an increase in Hgb level ≥2g/dL from baseline; OR
- Member has had normalization of Hgb level to ≥12g/dL; OR
- Member has had a decreased number of RBC transfusions since initiation of therapy.

Evrysdi™ (Risdiplam) Approval Criteria:

An FDA approved diagnosis of spinal muscular atrophy (SMA); AND
Molecular genetic testing to confirm bi-allelic pathogenic variants in the survival motor neuron 1 (SMN1) gene; AND
Member is not currently dependent on permanent invasive ventilation (defined as ≥16 hours of respiratory assistance per day continuously for >21 days in the absence of an acute, reversible illness or a perioperative state); AND
Evrysdi™ must be prescribed by a neurologist or specialist with expertise in the treatment of SMA (or an advanced care practitioner with a supervising physician who is a neurologist or specialist with expertise in the treatment of SMA); AND
Prescriber must agree to evaluate member’s liver function prior to initiating Evrysdi^® and must verify the member does not have severe hepatic impairment (Child-Pugh C); AND
Pharmacy must confirm Evrysdi™ will be constituted to an oral solution by a pharmacist prior to dispensing and must confirm Evrysdi™ will be shipped via cold chain supply to adhere to the storage and handling requirements in the Evrysdi™ Prescribing Information; AND
Prescriber must confirm the member or caregiver has been counseled on the proper storage of Evrysdi™ and has been instructed on how to prepare the prescribed daily dose of Evrysdi™ prior to administration of the first dose; AND
Female members of reproductive potential must not be pregnant and must have a negative pregnancy test prior to initiation of therapy; AND
Female members of reproductive potential must be willing to use effective contraception during treatment with Evrysdi™ and for at least 1 month after the last dose; AND
Prescriber must verify male members of reproductive potential have been counseled on the potential effects on fertility and the potential of compromised male fertility is acceptable; AND
Member will not be approved for concomitant treatment with Spinraza^® (nusinersen); AND
Member must not have previously received treatment with Zolgensma^® (onasemnogene abeparvovec-xioi); AND
A baseline assessment must be provided using a functionally appropriate exam [e.g., Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), Hammersmith Functional Motor Scale Expanded (HFMSE), Hammersmith Infant Neurological Exam (HINE), Upper Limb Module (ULM) Test]; AND
Initial authorizations will be for the duration of 6 months, at which time the prescriber must verify the member is compliant with Evrysdi™ and responding to the medication as demonstrated by clinically significant improvement or maintenance of function from pre-treatment baseline status using the same exam as performed at baseline assessment; AND
Member’s recent weight must be provided to ensure accurate dosing in accordance with Evrysdi™ Prescribing Information; AND
A quantity limit of 240mL per 36 days will apply.

Prior Authorization

amifampridine (Firdapse®) Approval Criteria:

A diagnosis of Lambert-Eaton myasthenic syndrome (LEMS); AND
Diagnosis must be confirmed by 1 of the following:
- A high titer anti-P/Q-type voltage-gated calcium channel (VGCC) antibody assay; OR
- A confirmatory electrodiagnostic study [e.g., repetitive nerve stimulation (RNS), needle electromyography (EMG), single-fiber electromyography (SFEMG)]; AND
Firdapse® must be prescribed by, or in consultation with, a neurologist or oncologist; AND
Member must not have a history of seizures or be taking medications that lower the seizure threshold (e.g., bupropion, tramadol, amphetamines, theophylline); AND
A quantity limit of 240 tablets per 30 days will apply; AND
Initial approvals will be for 6 months. Continued authorization will require the prescriber to indicate that the member is responding well to treatment and continues to require treatment with Firdapse®.

Prior Authorization form

emapalumab-lzsg (Gamifant®) Approval Criteria:

An FDA approved indication for the treatment of adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or who are intolerant to conventional HLH therapy; AND
Diagnosis of primary HLH must be confirmed by 1 of the following:
- Genetic testing confirming mutation of a gene known to cause primary HLH (e.g., PRF, UNC13D, STX11); OR
- Family history consistent with primary HLH; OR
- Member meets 5 of the following 8 diagnostic criteria:
  - Fever; OR
  - Splenomegaly; OR
  - Cytopenias affecting at least 2 of 3 lineages in the peripheral blood (hemoglobin <9, platelets <100 x 109/L, neutrophils <1 x 109/L); OR
  - Hypertriglyceridemia (fasting triglycerides >3mmol/L or ≥265mg/dL) and/or hypofibrinogenemia (≤1.5g/L); OR
  - Hemophagocytosis in bone marrow, spleen, or lymph nodes with no evidence of malignancy; OR
  - Low or absent natural killer (NK)-cell activity; OR
  - Hyperferritinemia (ferritin ≥500mcg/L); OR
  - High levels of soluble interleukin-2 receptor (soluble CD25 ≥2,400U/mL); AND
Gamifant® must be prescribed by, or in consultation with, a physician who specializes in the treatment of immune deficiency disorders; AND
Member must have at least 1 of the following:
- Failure of at least 1 conventional HLH treatment (e.g., etoposide, dexamethasone, cyclosporine); OR
- Documentation of progressive disease despite conventional HLH treatment; OR
- A patient-specific, clinically significant reason why conventional HLH treatment is not appropriate for the member must be provided; AND
Prescriber must verify dexamethasone dosed at least 5mg/m2/day will be used concomitantly with Gamifant®; AND
Prescriber must verify member has received or will receive prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infection(s); AND
Prescriber must verify member will be monitored for tuberculosis (TB), adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated; AND
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; AND
Approvals will be for the duration of 6 months with reauthorization granted if the prescriber documents the member is responding well to treatment, no unacceptable toxicity has occurred, and the member has not received hematopoietic stem cell transplantation (HSCT).

Prior Authorization form

Imcivree™ (Setmelanotide) Approval Criteria:

An FDA approved indication of chronic weight management in adult and pediatric members 6 years of age and older with obesity due to 1 of following:
- Proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency; OR
- Bardet-Biedl syndrome (BBS); AND
For POMC-, PCSK1-, or LEPR-deficiency, diagnosis must be confirmed by molecular genetic testing to confirm homozygous variants in the POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (results of genetic testing must be submitted); AND
Clinical features of BBS supported by detailed clinical documentation of each feature (medical records/clinical documentation of each feature must be submitted), diagnosis must be confirmed by the following:
- Molecular genetic testing to confirm homozygous variants in a BBS gene that are interpreted as pathogenic or likely pathogenic (results of genetic testing must be submitted); AND
- Four of the following primary features:
  - Rod-cone dystrophy, polydactyly, obesity, learning disabilities, hypogonadotropic hypogonadism and/or genitourinary anomalies, or renal anomalies; OR
- Three of the primary features previously listed in 3.b.i. plus 2 secondary features including:
  - Speech disorder/delay, strabismus/cataracts/astigmatism, brachydactyly/syndactyly, developmental delay, ataxia/poor coordination/imbalance, mild spasticity (especially lower limbs), diabetes mellitus, dental crowding/hypodontia/small roots/high arched palate, left ventricular hypertrophy/congenital heart disease, or hepatic fibrosis; AND
Requests for Imcivree™ for obesity due to suspected POMC-, PCSK1-, or LEPR-deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign or other types of obesity not related to POMC, PCSK1, or LEPR deficiency or BBS including obesity associated with other genetic syndromes, or general obesity will not be approved; AND
Member is currently on a dietician-guided diet and exercise program and has previously failed a dietician-guided diet and exercise program alone; AND
Member’s baseline weight and body mass index (BMI) must be provided; AND
Baseline BMI must be ≥30kg/m² for adults or ≥95^th percentile on BMI-for-age growth chart assessment for children; AND
Member must not be actively suicidal or have uncontrolled depression and prescriber must verify member will be monitored for depression prior to starting Imcivree™ therapy and throughout treatment; AND
Prescriber must verify member has been counseled on potential sexual adverse reactions and when to seek emergency medical care; AND
Prescriber must verify member does not have end stage renal disease [estimated glomerular filtration rate (eGFR) <15mL/min/1.73m²] and must confirm the dose will be adjusted per package labeling for members with severe renal impairment (eGFR 15 to 29mL/min/1.73m²); AND
Prescriber must verify female member is not pregnant or breastfeeding; AND
Prescriber must confirm member or caregiver has been trained on the proper storage and administration of Imcivree™ prior to the first dose; AND
For POMC-, PCSK1-, or LEPR-deficiency, initial approvals will be for the duration of 16 weeks. Reauthorization may be granted if the prescriber documents the member’s current weight or BMI and member has achieved weight loss of ≥5% of baseline body weight or ≥5% of BMI; OR
For BBS, approvals will be for the duration of 1 year. Reauthorization may be granted if the prescriber documents the member’s current weight or BMI and member has achieved weight loss of ≥5% of baseline body weight or ≥5% of BMI; AND
A quantity limit of 9mL per 30 days will apply.

Jesduvroq™ (Daprodustat) Approval Criteria:

An FDA approved indication for the treatment of anemia due to chronic kidney disease (CKD) in adults; and
Member must currently be on dialysis and must have been receiving dialysis for ≥4 months; and
Prescriber must verify that member does not have uncontrolled hypertension; and
Prescriber must verify that member does not have an active malignancy; and
Member must not be concurrently taking strong CYP2C8 inhibitors (i.e., gemfibrozil); and
Member’s pre-treatment hemoglobin (Hgb) must be <11g/dL. Recent Hgb levels must be provided; and
Member must be hyporesponsive to an erythropoiesis-stimulating agent (ESA) (or have a contraindication to use), defined as:
- No increase in Hgb after 1 month of weight-based dosing; or
- 2 increases in ESA dose up to 50% more than previous dose to maintain current Hgb level; and
Prescriber must verify that member will not use Jesduvroq™ concomitantly with an ESA; and
Initial and subsequent approvals will be for the duration of 12 weeks of treatment. Subsequent approvals will granted if the member meets 1 of the following:
- Member has achieved or maintained a clinically meaningful increase in Hgb of ≥1g/dL and the member’s Hgb level is <12g/dL; or
- If the member has not achieved or maintained a clinically meaningful increase in Hgb of ≥1g/dL, then all of the following will be required:
  - The dose will be increased as tolerated to a maximum of 24mg per day; and
  - The member has not received 24mg per day for >12 weeks without achieving a clinically meaningful increase in hemoglobin of ≥1g/dL; and
  - The member’s Hgb is <12g/dL; and
Jesduvroq™ should be discontinued in members who do not show evidence of a clinically meaningful increase in Hgb by 24 weeks.

Joenja^® (Leniolisib) Approval Criteria:

An FDA approved diagnosis of activated phosphoinositide 3-kinase (PI3K) delta syndrome (APDS). Diagnosis must be confirmed by the following:
- Genetic testing identifying a documented pathogenic variant in either the PIK3CD or PIK3R1 gene (results of genetic testing must be submitted); and
Member must be 12 years of age or older and weigh ≥45kg; and
Joenja^® must be prescribed by, or in consultation with, an immunologist, geneticist, or a specialist with expertise in treatment of APDS; and
Female members of reproductive potential must not be breastfeeding, must have a negative pregnancy test prior to initiation, and must agree to use effective contraception during treatment and for 1 week after the final dose of Joenja^®; and
Member must not have moderate to severe hepatic impairment (Child-Pugh class B or C); and
Member must not be taking any of the following medications concomitantly with Joenja^®:
- Strong CYP3A4 inhibitors (e.g., ketoconazole, itraconazole, clarithromycin); and
- Strong or moderate CYP3A4 inducers (e.g., rifampin, carbamazepine, phenytoin, St. John’s wort, phenobarbital, primidone); and
- CYP1A2 metabolized drugs with a narrow therapeutic range (e.g., tizanidine, theophylline); and
- OATP1B1/3 substrates (e.g., statins, bosentan, glyburide, nateglinide, repaglinide, methotrexate, furosemide); and
- BCRP transporter substrates (e.g., sulfasalazine, ubrogepant, tenofovir); and
Initial approvals will be for the duration of 3 months. Further approval may be granted if the prescriber documents the member is responding well to treatment; and
A quantity limit of 60 tablets per 30 days will apply.

Jynarque™ (Tolvaptan) Approval Criteria:

An FDA approved indication to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease (ADPKD); AND
Member must be 18 years of age or older; AND
Member must not have any contraindications to taking Jynarque™ including the following:
- Taking any concomitant strong CYP3A inhibitors (e.g., ketoconazole, itraconazole, lopinavir/ritonavir, indinavir/ritonavir, ritonavir, conivaptan); AND
- A history of signs or symptoms of significant liver impairment or injury (does not include uncomplicated polycystic liver disease); AND
- Uncorrected abnormal blood sodium concentrations; AND
- Unable to sense or respond to thirst; AND
- Hypovolemia; AND
- Hypersensitivity to tolvaptan or any of its components; AND
- Uncorrected urinary outflow obstruction; AND
- Anuria; AND
Member must not be taking any of the following medications concomitantly with Jynarque™:
- Strong CYP3A inhibitors (e.g., ketoconazole, itraconazole, lopinavir/ritonavir, indinavir/ritonavir, ritonavir, conivaptan); AND
- Strong CYP3A inducers (e.g., rifampin); AND
- OATP1B1/3 and OAT3 transporter substrates (e.g., statins, bosentan, glyburide, nateglinide, repaglinide, methotrexate, furosemide); AND
- BCRP transporter substrates (e.g., rosuvastatin); AND
- V2-receptor agonists (e.g., desmopressin); AND
Jynarque™ must be prescribed by a nephrologist or specialist with expertise in the treatment of ADPKD (or be an advanced care practitioner with a supervising physician who is a nephrologist or specialist with expertise in the treatment of ADPKD); AND
Prescriber must agree to assess ALT, AST, and bilirubin prior to initiation of Jynarque™, at 2 weeks and 4 weeks after initiation, then monthly for 18 months, and every 3 months thereafter; AND
Female members must not be pregnant and must have a negative pregnancy test prior to therapy initiation; AND
Prescriber, pharmacy, and member must be enrolled in the Jynarque™ Risk Evaluation and Mitigation Strategy (REMS) program and maintain enrollment throughout therapy.

Prior Authorization form

Livmarli™ (Maralixibat) Approval Criteria:

An FDA approved indication for the treatment of cholestatic pruritus in members with Alagille Syndrome (ALGS); and
- Diagnosis must be confirmed by genetic testing identifying mutations in the JAG1 or NOTCH2 genes; and
Member must be 1 year of age or older; and
Livmarli™ must be prescribed by a gastroenterologist, hepatologist, geneticist, or other specialist with expertise in the treatment of ALGS (or an advanced care practitioner with a supervising physician who is a gastroenterologist, hepatologist, geneticist, or other specialist with expertise in the treatment of ALGS); and
Prescriber must verify member has a history of significant pruritus that is unresponsive to treatment with ursodeoxycholic acid (UDCA) and at least 2 of the following, unless contraindicated:
- Cholestyramine; or
- Rifampin; or
- Sertraline; or
- Naltrexone; and
Member must have evidence of cholestasis demonstrated by ≥1 of the following:
- Total serum bile acid >3x upper limit of normal (ULN) for age; or
- Conjugated bilirubin >1mg/dL; or
- Fat soluble vitamin deficiency otherwise unexplainable; or
- Gamma-glutamyl transferase (GGT) >3x ULN for age; or
- Intractable pruritus explainable only by liver disease; and
Members with a history of liver transplantation will not generally be approved for Livmarli™; and
Prescriber must verify surgical intervention (e.g., biliary diversion, liver transplantation) is not currently clinically appropriate for the member; and
Prescriber must agree to monitor alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin, direct bilirubin, and international normalized ratio (INR) at baseline and during treatment with Livmarli™; and
Prescriber must verify the member and/or member’s caregiver has been counseled on appropriate storage, dosing, and administration of Livmarli™, including the use of a calibrated oral dosing dispenser for accurate measurement; and
Member’s current weight (taken within the past 3 weeks) must be provided on initial and subsequent prior authorization requests in order to authorize the appropriate amount of drug required according to package labeling; and
Initial approvals will be for a duration of 3 months. After 3 months of treatment, further approval may be granted for a duration of 1 year if the prescriber documents the member is responding well to treatment and surgical intervention is still not clinically appropriate.

Luxturna™ (Voretigene Neparvovec-rzyl) Approval Criteria:

An FDA approved diagnosis of biallelic RPE65 mutation-associated retinal dystrophy; AND
- Diagnosis must be confirmed by genetic testing; AND
Member must have sufficient viable retinal cells in both eyes as determined by the treating physician(s); AND
Member must have best corrected visual acuity of 20/60 or worse in both eyes and/or visual field less than 20 degrees in any meridian in both eyes; AND
Member must be four years of age or older; AND
Member must not have participated in a previous RPE65 gene therapy study or have previously received treatment with Luxturna™; AND
Member must not have had intraocular surgery in the past 6 months; AND
Female members of child bearing age must not be pregnant and must have a negative pregnancy test immediately prior to administration of Luxturna™; AND
Male and female members of child bearing age must be willing to use effective contraception during treatment with Luxturna™ and for at least 4 months after administration of Luxturna™; AND
Member must take the recommended systemic oral corticosteroid regimen, starting 3 days prior to administration of Luxturna™ to each eye, and continuing after administration of Luxturna™, as per package labeling of Luxturna™; AND
Luxturna™ must be prescribed and administered by a retinal surgeon with expertise in the treatment of biallelic RPE65 mutation-associated retinal dystrophy and in the administration of Luxturna™ at an Ocular Gene Therapy Treatment Center; AND
- Luxturna™ must be shipped via cold chain supply shipping and delivery to the Ocular Gene Therapy Treatment Center where the member is scheduled to receive treatment; AND
- Luxturna™ must be stored frozen prior to preparation for administration (Luxturna™ should be administered within 4 hours of preparation); AND
- The receiving facility must have in place a mechanism to track patient-specific Luxturna™ from receipt to storage to administration; AND
Luxturna™ must be administered subretinally to each eye on separate days within a close interval, but no fewer than 6 days apart; AND
- The scheduled procedure date for each eye must be provided; AND
Only one single-dose vial per eye will be approved per member per lifetime; AND
- Each single-dose vial of Luxturna™ is to be dispensed immediately prior to the scheduled procedure for the specific eye.
A prior authorization request with patient-specific information may be submitted for consideration of Luxturna™ for members not meeting all of the current prior authorization criteria requirements.

Outpatient Medical Petition

Aldurazyme^® (Laronidase) Approval Criteria:

An FDA approved diagnosis of Hurler, Hurler-Scheie, or Scheie syndrome (mucopolysaccharidosis type I; MPS I) confirmed by:
- Enzyme assay demonstrating a deficiency of alpha-L-iduronidase (IDUA) enzyme activity (results of assay must be submitted); or
- Molecular genetic testing to confirm biallelic pathogenic mutations in the IDUA gene (results of genetic testing must be submitted); and
For Scheie syndrome, the prescriber must document that the member has moderate-to-severe symptoms; and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of MPS I; and
Aldurazyme^® must be administered by a health care professional prepared to manage anaphylaxis; and
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; and
Initial approvals will be for the duration of 6 months. Reauthorization may be granted if the prescriber documents the member is responding well to treatment. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Brineura^® (Cerliponase Alfa) Approval Criteria:

An FDA approved diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) also known as tripeptidyl peptidase-1 (TPP-1) deficiency confirmed by:
- Enzyme assay demonstrating a deficiency of TPP-1 enzyme activity (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the TPP1 gene (results of genetic testing must be submitted); and
Member must be 3 years of age or older; and
Brineura^® must be prescribed by a specialist with expertise in the treatment of CLN2 (or an advanced care practitioner with a supervising physician who is a specialist with expertise in treating CLN2); and
Brineura^® must be administered in a health care facility by a prescriber who is knowledgeable in intraventricular administration; and
Member must not have ventriculoperitoneal shunts or acute intraventricular access device-related complications; and
Member must not have documented generalized status epilepticus within 4 weeks of initiating treatment; and
Prescriber must verify member’s blood pressure and heart rate will be monitored prior to each infusion, during infusion, and post-infusion; and
Prescriber must be willing to perform regular 12-lead electrocardiogram (ECG) evaluation at baseline and at least every 6 months and verify that they are acceptable to the prescriber; and
A baseline assessment must be performed to assess the Motor plus Language CLN2 score; and
Initial authorizations will be for the duration of 6 months, at which time compliance will be required for continued approval. After 12 months of utilization, the prescriber must verify the member is responding to the medication as demonstrated by ≤2 point decline in Motor plus Language CLN2 score from baseline. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment; and
Approval quantity will be based on package labeling and FDA approved dosing regimen.

Cerdelga^® (Eliglustat) Approval Criteria:

An FDA approved diagnosis of type 1 Gaucher disease (GD1) confirmed by:
- Enzyme assay demonstrating a deficiency of glucocerebrosidase enzyme activity (≤15% of normal) (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the GBA1 gene (results of genetic testing must be submitted); and
Member is classified as 1 of the following as detected by an FDA-cleared test:
- CYP2D6 extensive metabolizers (EMs); or
- CYP2D6 intermediate metabolizers (IMs); or
- CYP2D6 poor metabolizers (PMs); and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of GD1; and
Prescriber must verify the member will not take Cerdelga^® concurrently with another therapy for GD1; and
For CYP2D6 EMs and IMs, a quantity limit of 56 capsules per 28 days will apply. For CYP2D6 PMs, a quantity limit of 28 capsules per 28 days will apply; and
Initial approvals will be for the duration of 6 months, at which time the prescriber must verify the member is responding well to the medication. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Cerezyme^® (Imiglucerase), Elelyso^® (Taliglucerase Alfa), and Vpriv^® (Velaglucerase Alfa) Approval Criteria:

An FDA approved diagnosis of Gaucher disease (GD) confirmed by:
- Enzyme assay demonstrating a deficiency of glucocerebrosidase enzyme activity (≤15% of normal) (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the GBA1 gene (results of genetic testing must be submitted); and
Prescriber must confirm member has symptomatic (e.g., anemia, thrombocytopenia, bone disease, splenomegaly, hepatomegaly) type 1 or type 3 GD; and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of GD; and
Member’s weight (kg) must be provided and must have been taken within the last 4 weeks to ensure accurate weight-based dosing; and
Prescriber must verify the member will not take the requested therapy concurrently with another therapy for GD; and
Initial approvals will be for the duration of 6 months, at which time the prescriber must verify the member is responding well to the medication. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Elaprase^® (Idursulfase) Approval Criteria:

An FDA approved diagnosis of Hunter syndrome (mucopolysaccharidosis type II; MPS II) confirmed by:
- Enzyme assay demonstrating a deficiency of iduronate-2-sulfatase enzyme activity (results of assay must be submitted); or
- Molecular genetic testing confirming a hemizygous pathogenic variant in the IDS gene (results of genetic testing must be submitted); and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of MPS II; and
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; and
Initial approvals will be for the duration of 6 months, at which time the prescriber must verify the member is responding well to the medication. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Elfabrio^® (Pegunigalsidase Alfa-iwxj) and Fabrazyme^® (Agalsidase Beta) Approval Criteria:

An FDA approved diagnosis of Fabry disease confirmed by 1 of the following:
- Molecular genetic testing confirming a pathogenic variant in the galactosidase alpha (GLA) gene (results of genetic testing must be submitted); or
- Enzyme assay demonstrating a deficiency of alpha-galactosidase A enzyme activity (<5% of normal) (results of assay must be submitted); and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of Fabry disease; and
Requests for Elfabrio^® will require a patient-specific, clinically significant reason why the member cannot use Fabrazyme^®; and
Member will not be approved for concomitant use with Galafold^® (migalastat); and
Member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; and
Initial approvals will be for the duration of 6 months. After that time, compliance will be required for continued authorization and prescriber must verify the member is responding well to treatment. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Galafold^® (Migalastat) Approval Criteria:

An FDA approved diagnosis of Fabry disease with a confirmed amenable galactosidase alpha (GLA) gene variant based on in vitro assay data (results of genetic testing must be submitted); and
Galafold^® must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of Fabry disease (or an advanced care practitioner with a supervising physician who is a geneticist or other specialist with expertise in the treatment of Fabry disease); and
Member must have an estimated glomerular filtration rate (eGFR) of ≥30mL/min/1.73m²; and
Galafold^® will not be approved for concomitant use with enzyme replacement therapy (ERT); and
Galafold^® will initially be approved for 6 months. After that time, compliance will be required for continued approval and prescriber must verify the member is responding well to treatment. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment; and
A quantity limit of 14 capsules per 28 days will apply.

Kanuma^® (Sebelipase Alfa) Approval Criteria:

An FDA approved diagnosis of lysosomal acid lipase (LAL) deficiency confirmed by:
- Enzyme assay demonstrating a deficiency of LAL enzyme activity (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the LIPA gene (results of genetic testing must be submitted); and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of LAL deficiency; and
Kanuma^® (sebelipase alfa) must be administered in a health care setting by a health care professional prepared to manage anaphylaxis; and
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; and
Initial approvals will be for the duration of 6 months, at which time the prescriber must verify the member is responding well to the medication. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Lamzede^® (Velmanase Alfa-tycv) Approval Criteria:

An FDA approved diagnosis of alpha-mannosidosis confirmed by:
- Enzyme assay verifying alpha-mannosidase enzyme activity <11% of normal (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the MAN2B1 gene (results of genetic testing must be submitted); and
Member’s recent weight (kg) taken within the last 3 weeks must be provided to ensure accurate weight-based dosing; and
Female members of reproductive potential must have a negative pregnancy test prior to initiation and must agree to use effective contraception during treatment and for 2 weeks after the final dose of Lamzede^®; and
Lamzede^® must be administered in a health care setting by a health care provider with appropriate equipment and personnel to manage anaphylaxis. Approvals will not be granted for self-administration; and
- Lamzede^® must be shipped via cold chain supply to the health care setting where the member is scheduled to receive treatment; and
Lamzede^® must be prescribed by, or in consultation with, a specialist with expertise in the treatment of lysosomal storage disorders; and
Initial approvals will be for the duration of 6 months. Further approval may be granted if the prescriber documents the member is responding well to treatment. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Lumizyme^® (Alglucosidase Alfa) Approval Criteria [Infantile-Onset Pompe Disease Diagnosis]:

An FDA approved diagnosis of infantile-onset Pompe disease [acid alpha-glucosidase (GAA) deficiency] confirmed by:
- Enzyme assay demonstrating a deficiency of GAA enzyme activity (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the GAA gene (results of genetic testing must be submitted); and
Lumizyme^® must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of Pompe disease and/or inherited genetic disorders; and
Member’s weight must be provided and have been taken within the last 4 weeks to ensure accurate dosing.

Lumizyme^® (Alglucosidase Alfa) Approval Criteria [Late-Onset (Non-Infantile) Pompe Disease Diagnosis]:

An FDA approved diagnosis of late-onset (non-infantile) Pompe disease [acid alpha-glucosidase (GAA) deficiency] confirmed by:
- Enzyme assay demonstrating a deficiency of GAA enzyme activity (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the GAA gene (results of genetic testing must be submitted); and
Provider must document presence of symptoms of Pompe disease; and
Lumizyme^® must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of Pompe disease and/or inherited genetic disorders; and
Member’s weight must be provided and have been taken within the last 4 weeks to ensure accurate dosing; and
Initial approval will be for the duration of 6 months, at which time compliance and information regarding efficacy, such as improvement or stabilization in forced vital capacity (FVC) and/or 6-minute walk test (6MWT), will be required for continued approval. Subsequent approvals will be for the duration of 1 year.

Mepsevii^® (Vestronidase Alfa-vjbk) Approval Criteria:

An FDA approved diagnosis of Sly syndrome (mucopolysaccharidosis VII; MPS VII) confirmed by:
- Enzyme assay demonstrating a deficiency of beta-glucuronidase enzyme activity (results of assay must be submitted); or
- Molecular genetic testing to confirm biallelic pathogenic variants in the GUSB gene (results of genetic testing must be submitted); and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of MPS VII; and
Mepsevii^® must be administered by a health care professional prepared to manage anaphylaxis; and
Initial approvals will be for the duration of 12 months. Reauthorization may be granted if the prescriber documents the member is responding well to treatment; and
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling.

Naglazyme^® (Galsulfase) Approval Criteria:

An FDA approved diagnosis of Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI; MPS VI) confirmed by:
- Enzyme assay demonstrating a deficiency of arylsulfatase B (ASB) enzyme activity (results of assay must be submitted); or
- Molecular genetic testing to confirm biallelic pathogenic variants in the ARSB gene (results of genetic testing must be submitted); and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of MPS VI; and
Naglazyme^® must be administered by a health care professional prepared to manage anaphylaxis; and
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; and
Initial approvals will be for the duration of 6 months. Reauthorization may be granted if the prescriber documents the member is responding well to treatment. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Nexviazyme^® (Avalglucosidase Alfa-ngpt) Approval Criteria:

An FDA approved diagnosis of late-onset (non-infantile) Pompe disease [acid alpha-glucosidase (GAA) deficiency] confirmed by:
- Enzyme assay demonstrating a deficiency of GAA enzyme activity (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the GAA gene (results of genetic testing must be submitted); and
Prescriber must document presence of symptoms of Pompe disease; and
Nexviazyme^® must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of Pompe disease and/or inherited genetic disorders; and
Member’s weight must be provided and have been taken within the last 4 weeks to ensure accurate dosing; and
Initial approval will be for the duration of 6 months, at which time compliance and information regarding efficacy, such as improvement or stabilization in forced vital capacity (FVC) and/or 6-minute walk test (6MWT), will be required for continued approval. Subsequent approvals will be for the duration of 1 year.

Opfolda™ (Miglustat) and Pombiliti™ (Cipaglucosidase Alfa-atga) Approval Criteria:

An FDA approved diagnosis of late-onset (non-infantile) Pompe disease [acid alpha-glucosidase (GAA) deficiency] confirmed by:
- Enzyme assay demonstrating a deficiency of GAA enzyme activity (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the GAA gene (results of genetic testing must be submitted); and
Member must be 18 years of age or older and weigh ≥40kg; and
Prescriber must document presence of symptoms of Pompe disease; and
Member must be receiving a different enzyme replacement therapy (ERT) for Pompe disease and not experiencing improvement on the current ERT product; and
Female members of reproductive potential must have a negative pregnancy test prior to initiation and must agree to use effective contraception during treatment and for at least 60 days after the final dose; and
Pombiliti™ must be administered in a health care setting by a health care provider with appropriate equipment and personnel to manage anaphylaxis. Approvals will not be granted for self administartion; and
- Must be shipped via cold chain supply to the health care setting where the member is scheduled to receive treatment; and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of Pompe disease; and
Opfolda™ must be used in combination with Pombiliti™; and
- A separate, completed prior authorization request must be received for both medications; and
Member will not be approved for concomitant use with other ERT products for Pompe disease; and
Member’s recent weight must be provided in order to authorize the appropriate amount of drug required according to package labeling; and
For Opfolda™, the following quantity limits will apply;
- Weight ≥50kg: 8 capsules per 28 days; or
- Weight 40 kg to <50kg: 6 capsules per 28 days; and
Initial approvals will be for the duration of 6 months, at which time compliance and information regarding efficacy, such as improvement or stabilization in forced vital capacity (FVC) and/or 6-minute walk test (6MWT), will be required for continued approval. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Procysbi^® (Cysteamine Bitartrate) Delayed-Release Capsule and Granule Approval Criteria:

An FDA approved diagnosis of nephropathic cystinosis confirmed by 1 of the following:
- Identification of elevated cystine concentration in polymorphonuclear leukocytes; or
- Molecular genetic testing confirming biallelic pathogenic variants in the CTNS gene (results of genetic testing must be submitted); and
Must be prescribed by, or in consultation with, a nephrologist or other specialist with expertise in the treatment of cystinosis; and
A patient specific, clinically significant reason why the member cannot use the short-acting formulation, Cystagon^® (cysteamine bitartrate), must be provided; and
Use of Procysbi^® granules will also require a patient specific, clinically significant reason why the member cannot use the capsule formulation of Procysbi^®.

Vimizim^® (Elosulfase Alfa) Approval Criteria:

An FDA approved diagnosis of Morquio A syndrome (mucopolysaccharidosis type IVA; MPS IVA) confirmed by:
- Enzyme assay demonstrating a deficiency of N-acetylgalactosamine-6-sulfatase (GALNS) enzyme activity (results of assay must be submitted); or
- Molecular genetic testing to confirm biallelic pathogenic variants in the GALNS gene (results of genetic testing must be submitted); and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of MPS IVA; and
Vimizim^® must be administered by a health care professional prepared to manage anaphylaxis; and
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; and
Initial approvals will be for the duration of 12 months. Reauthorization may be granted if the prescriber documents the member is responding well to treatment.

Xenpozyme^® (Olipudase Alfa-rpcp) Approval Criteria:

An FDA approved diagnosis of acid sphingomyelinase deficiency (ASMD) type B or A/B confirmed by:
- Documented lab results verifying <10% of acid sphingomyelinase (ASM) activity from control (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the SMPD1 gene (results of genetic testing must be submitted); and
Documentation of baseline AST and ALT within 1 month prior to treatment initiation or within 72 hours prior to treatment escalation; and
Member’s weight (kg) and body mass index (BMI) within the last 3 weeks must be provided to ensure accurate weight-based dosing; and
- BMI ≤30: The dosage is based on actual body weight (kg); or
- BMI >30: The dosage is based on adjusted body weight; and
Female members of reproductive potential must have a negative pregnancy test prior to initiation and must agree to use effective contraception during treatment and for 2 weeks after the final dose of Xenpozyme^®; and
Prescriber must verify ALT and AST will be assessed to manage the risk of elevated transaminases as directed by package labeling; and
Xenpozyme^® must be administered by a health care provider prepared to manage anaphylaxis. Approvals will not be granted for self-administration. Prior authorization requests must indicate how Xenpozyme^® will be administered; and
- Xenpozyme^® must be shipped via cold chain supply to the health care facility where the member is scheduled to receive treatment; or
- Xenpozyme^® must be shipped via cold chain supply to the member’s home and administered by a home health care provider prepared to manage anaphylaxis, and the member or member’s caregiver must be trained on the proper storage of Xenpozyme^®; and
  - For consideration of home administration by a home health care provider, prescriber must verify member is receiving the maintenance dose and is tolerating the Xenpozyme^® infusion well; and
Xenpozyme^® must be prescribed by, or in consultation with, a specialist with expertise in the treatment of lysosomal storage disorders; and
Initial approvals will be for the duration of 6 months. Further approval may be granted if the prescriber documents that the member is responding well to treatment. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Zavesca^® (Miglustat) Approval Criteria:

An FDA approved diagnosis of mild/moderate type 1 Gaucher disease (GD1) confirmed by:
- Enzyme assay demonstrating a deficiency of glucocerebrosidase enzyme activity (≤15% of normal) (results of assay must be submitted); or
- Molecular genetic testing confirming biallelic pathogenic variants in the GBA1 gene (results of genetic testing must be submitted); and
A patient-specific, clinically significant reason why the member cannot use 1 of the following enzyme replacement therapies must be provided:
- Cerezyme^® (imiglucerase); or
- Elelyso^® (taliglucerase alfa); or
- Vpriv^® (velaglucerase alfa); and
Zavesca^® is brand preferred. Requests for generic miglustat will require a patient-specific, clinically significant reason why the member cannot use the brand formulation; and
Must be prescribed by, or in consultation with, a geneticist or other specialist with expertise in the treatment of GD1; and
Prescriber must verify the member will not take Zavesca^®concurrently with another therapy for GD1; and
A quantity limit of 90 capsules per 30 days will apply; and
Initial approvals will be for the duration of 6 months, at which time the prescriber must verify the member is responding well to the medication. Subsequent approvals will be for the duration of 1 year if the member is responding well to treatment.

Nulibry™ (Fosdenopterin) Approval Criteria:

An FDA approved indication to reduce the risk of mortality in members with molybdenum cofactor deficiency (MoCD) Type A; and
MoCD Type A must be confirmed by genetic testing; and
a. If the member is presumed to have MoCD Type A, Nulibry™ can be approved for 1 month until genetic testing can be performed; and
b. Nulibry™ will be discontinued if genetic testing results do not confirm MoCD Type A; and
Nulibry™ must be administered by a health care provider or the prescriber must verify the member or member’s caregiver has been trained by a health care professional on proper storage, preparation, and intravenous (IV) administration of Nulibry™; and
Member’s weight (kg) must be provided and must have been taken within the last 4 weeks to ensure accurate weight-based dosing according to package labeling; and
Approval quantities will be dependent on the member’s age, weight, and dosing based on the Nulibry™ Prescribing Information.

An FDA approved indication for the treatment of sickle cell disease (SCD) in members 4 years of age and older; AND
Member must have baseline hemoglobin ≤10.5g/dL; AND
Oxbryta® must be prescribed by, or in consultation with, a hematologist or a specialist with expertise in treatment of SCD (or an advanced care practitioner with a supervising physician who is a hematologist or specialist with expertise in treating SCD); AND
Prescriber must verify that the dose of Oxbryta® will be reduced in accordance with package labeling for members with severe hepatic impairment; AND
The member must not be taking concomitant strong or moderate CYP3A4 inducers (e.g., rifampin) or the prescriber must verify the dose of Oxbryta® will be adjusted during concomitant use according to package labeling; AND
For members younger than 12 years of age, the member’s recent weight (kg) must be provided on the prior authorization request to ensure accurate dosing in accordance with Oxbryta^® Prescribing Information; AND
Oxbryta^® tablets for oral suspension will have an age restriction of 4 to 10 years of age; AND
- Members older than 10 years of age requesting Oxbryta^® tablets for oral suspension will require a patient-specific, clinically significant reason why the member cannot use Oxbryta^® oral tablets; AND
The following quantity limits will apply :
- (3) 500mg tablets per day; AND
- (5) 300mg tablets for oral suspension per day; AND
Initial approvals will be for the duration of 6 months. Subsequent approvals will be for 1 year if the prescriber documents the member is responding well to treatment.

Oxlumo™ (Lumasiran) Approval Criteria:

An FDA approved indication for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels. Diagnosis of PH1 must be confirmed by:
- Molecular genetic testing identifying biallelic pathogenic variants in the AGXT gene (results of genetic testing must be submitted); or
- Liver biopsy confirming alanine-glyoxylate aminotransferase (AGT) catalytic deficiency if the results of genetic testing are not diagnostic (results of liver biopsy must be submitted); and
Oxlumo™ must be prescribed by a nephrologist, geneticist, or other specialist with expertise in the treatment of PH1 (or an advanced care practitioner with a supervising physician who is a nephrologist, geneticist, or other specialist with expertise in the treatment of PH1); and
The member must not have a history of liver transplant; and
The prescriber must verify that Oxlumo™ will be administered by a health care professional; and
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to the Oxlumo™ Prescribing Information; and
Initial approvals will be for the duration of 6 months. Further approval may be granted if the prescriber documents that the member is responding well to treatment as indicated by a reduction in urinary oxalate excretion or plasma oxalate levels.

Pyrukynd^® (Mitapivat) Approval Criteria:

An FDA approved indication of hemolytic anemia in adults with pyruvate kinase (PK) deficiency confirmed by the following:
- Presence of at least 2 variant alleles in the pyruvate kinase liver and red blood cell (PKLR) gene, with at least 1 missense variant; AND
  - Hemoglobin (Hgb) ≤10g/dL; OR
  - Member has received ≥6 red blood cell (RBC) transfusions in the past year; AND
Pyrukynd^® must be prescribed by a hematologist (or an advanced care practitioner with a supervising physician who is a hematologist); AND
Member must not have moderate or severe hepatic impairment; AND
If Pyrukynd^® is to be discontinued, prescriber must verify dose will be tapered gradually according to Pyrukynd^® Prescribing Information and member will be monitored for signs of acute hemolysis and worsening anemia; AND
Prescriber must agree to monitor Hgb levels and follow dose titration and maintenance according to Pyrukynd^® Prescribing Information; AND
Approvals will be for the duration of 6 months, after which time the prescriber must provide Hgb levels to support a dose increase or continuation of current dose; AND
Pyrukynd^® should be discontinued in members who do not show evidence of therapeutic benefit (i.e., Hgb increase of ≥1mg/dL from baseline, reduction in number of transfusions, improvement in hemolysis laboratory assessments) by week 24. Members will be granted short term approval to allow for gradual tapering per package labeling.

Reblozyl® (Luspatercept-aamt) Approval Criteria [Beta Thalassemia Diagnosis]:

An FDA approved indication for the treatment of adult members with beta thalassemia who require regular red blood cell (RBC) transfusions; AND
Member must require regular RBC transfusions (no transfusion-free period >35 days during the prior 6 month period); AND
Member must not have previously received treatment with Zynteglo^® (betibeglogene autotemcel); AND
Reblozyl® must be prescribed by, or in consultation with, a hematologist or a specialist with expertise in treatment of beta thalassemia (or an advanced care practitioner with a supervising physician who is a hematologist or specialist with expertise in treating beta thalassemia); AND
The prescriber must verify the member’s hemoglobin will be monitored prior to each Reblozyl® administration; AND
Prescriber must verify Reblozyl® will be administered by a trained health care provider; AND
A recent (within the last 3 months) weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; AND
Approval quantities will be dependent on member weight and every 3 week dosing in accordance with package labeling; AND
Initial approvals will be for the duration of 4 months. Further approvals will not be granted if the member does not experience a decrease in transfusion burden after 9 weeks of treatment (administration of 3 doses) at the maximum dose of 1.25mg/kg (allows for initial dosing of 6 weeks at 1mg/kg). Subsequent approvals will be for 1 year if the prescriber documents the member is responding well to treatment.

Reblozyl® (Luspatercept-aamt) Approval Criteria [Myelodysplastic Syndromes (MDS) Diagnosis]:

An FDA approved indication of 1 of the following:
- Treatment of adult members with very low-to-intermediate risk MDS with ring sideroblasts (MDS-RS) or myelodysplastic/ myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) with anemia failing an erythropoiesis-stimulating agent (ESA) and requiring ≥2 red blood cell (RBC) units over 8 weeks; OR
- Treatment of adult members with very low-to-intermediate risk MDS with anemia who are ESA-naïve and who required ≥2 RBC units within the last 8 weeks; AND
For MDS-RS or MDS/MPN-RS-T:
- Member must have had an inadequate response to prior treatment with an ESA, be intolerant of ESAs, or have a serum erythropoietin level >200U/L; AND
- Member must not have been previously treated with a disease modifying agent for the treatment of MDS; AND
- Prescriber must verify the member does not have deletion 5q (del 5q); AND
Complete blood counts (CBC) and verification that levels are acceptable to the prescriber and in accordance with package labeling; AND
Reblozyl® must be prescribed by, or in consultation with, a hematologist, oncologist, or a specialist with expertise in treatment of MDS (or an advanced care practitioner with a supervising physician who is a hematologist, oncologist, or specialist with expertise in treating MDS); AND
The prescriber must verify the member’s hemoglobin will be monitored prior to each Reblozyl® administration; AND
Prescriber must verify Reblozyl® will be administered by a trained health care provider; AND
A recent (within the last 3 months) weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; AND
Approval quantities will be dependent on member weight and every 3 week dosing in accordance with package labeling; AND
Initial approvals will be for the duration of 6 months. Further approvals will not be granted if the member does not experience a decrease in transfusion burden after 9 weeks of treatment (administration of 3 doses) at the maximum dose of 1.75mg/kg or if unacceptable toxicity occurs at any time. Subsequent approvals will be for 1 year if the prescriber documents the member is responding well to treatment.

Revcovi™ (Elapegademase-lvlr) Approval Criteria:

An FDA approved diagnosis of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients; AND
- Diagnosis of ADA deficiency should be confirmed by demonstrating biallelic mutations in the ADA gene; AND
Revcovi™ must be prescribed by or in consultation with a physician who specializes in the treatment of immune deficiency disorders; AND
The member must have failed to respond to a bone marrow transplant or not be a current suitable candidate for a bone marrow transplant; AND
A patient-specific, clinically significant reason why Adagen® (pegademase bovine) is not appropriate for the member; OR
Previous failure of Adagen® (pegademase bovine) used compliantly. Failure is defined as the inability to maintain ADA activity or reduce erythrocyte deoxyadenosine nucleotides (dAXP), or the member is experiencing adverse effects associated with Adagen® therapy that are not expected to occur with Revcovi™; AND
Prescriber must agree to monitor trough plasma ADA activity, trough dAXP levels, and/or total lymphocyte counts to ensure efficacy and compliance and to monitor for neutralizing antibodies when suspected; AND
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; AND
Initial approvals will be for the duration of 6 months at which time the prescriber must confirm improvement or stabilization in ADA activity or dAXP levels or improvement in immune function. Subsequent approvals will require the prescriber to verify the member is still not a current suitable candidate for a bone marrow transplant.

Prior Authorization form

amifampridine (Firdapse®) Approval Criteria:

A diagnosis of Lambert-Eaton myasthenic syndrome (LEMS); AND
Diagnosis must be confirmed by 1 of the following:
- A high titer anti-P/Q-type voltage-gated calcium channel (VGCC) antibody assay; OR
- A confirmatory electrodiagnostic study [e.g., repetitive nerve stimulation (RNS), needle electromyography (EMG), single-fiber electromyography (SFEMG)]; AND
Firdapse® must be prescribed by, or in consultation with, a neurologist or oncologist; AND
Member must not have a history of seizures or be taking medications that lower the seizure threshold (e.g., bupropion, tramadol, amphetamines, theophylline); AND
For Firdapse®, a patient-specific, clinically significant reason why the member cannot use Ruzurgi® must be provided; AND
For Firdapse®, a quantity limit of 240 tablets per 30 days will apply. For Ruzurgi®, a quantity limit of 300 tablets per 30 days will apply; AND
Initial approvals will be for 6 months. Continued authorization will require the prescriber to indicate that the member is responding well to treatment and continues to require treatment with Firdapse®.

Prior Authorization form

Ryplazim^® (Plasminogen, Human-tvmh) Approval Criteria:

An FDA approved indication of plasminogen deficiency type 1 (hypoplasminogenemia) as confirmed by at least 2 of the following:
- Genetic testing confirming biallelic mutations in the plasminogen (PLG) gene; or
- Plasminogen activity level ≤45%; or
- Documentation of clinical symptoms and lesions consistent with plasminogen deficiency type 1 (e.g., ligneous conjunctivitis, ligneous gingivitis or gingival overgrowth, vision abnormalities, respiratory distress and/or obstruction, abnormal wound healing); and
Ryplazim^® must be prescribed by, or in consultation with, a hematologist, pulmonologist, ophthalmologist, geneticist, or other specialist with expertise in the treatment of plasminogen deficiency (or an advanced care practitioner with a supervising physician who is a hematologist, pulmonologist, ophthalmologist, geneticist, or other specialist with expertise in the treatment of plasminogen deficiency); and
Prescriber must verify that members at high risk for bleeding and/or confirmed or suspected airway disease will be monitored by a health care provider for 4 hours after receiving the first dose; and
Documented vaccination history to hepatitis A and B must be provided or provider must verify member has received the first vaccine dose and is scheduled to receive the second vaccine dose; and
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; and
Initial approvals will be for 6 months, after which time the prescriber must document improvement in clinical symptoms, partial or complete lesion resolution, and increased plasminogen activity level; and
Subsequent approvals will be for the duration of 1 year and will require documentation from the prescriber that member has not developed new or recurrent lesions while on Ryplazim^® and that adequate plasminogen activity trough levels are being maintained.

hydroxyurea tablets(Siklos®) Approval Criteria:

An FDA approved indication of sickle cell anemia; AND
Member must be 2 years of age or older; AND
Member must have a history of moderate-to-severe, painful crises; AND
A trial of hydroxyurea capsules or a patient-specific, clinically significant reason why hydroxyurea capsules are not appropriate for the member; AND
Prescriber must agree to monitor blood counts every 2 weeks throughout therapy; AND
Prescriber must agree to monitor the member for the development of secondary malignancies; AND
Female members must not be pregnant and must have a negative pregnancy test prior to therapy initiation; AND
Male and female members of reproductive potential must be willing to use effective contraception during and after treatment with Siklos® for at least 6 months after therapy; AND
Initial approvals will be for the duration of 12 months. Reauthorization may be granted if the prescriber documents the member is responding well to treatment.

Prior Authorization form

Skyclarys™ (Omaveloxolone) Approval Criteria:

An FDA approved diagnosis of Friedreich’s ataxia (FRDA); AND
- Diagnosis must be confirmed by genetic testing identifying biallelic pathogenic variants in the frataxin (FXN) gene; AND
Member must be 16 years of age or older; AND
Skyclarys™ must be prescribed by, or in consultation with, a neurologist (or an advanced care practitioner with a supervising physician who is a neurologist); AND
Member must have a left ventricular ejection fraction of ≥40%; AND
Member must not be taking concomitant strong or moderate CYP3A4 inhibitors (e.g., ketoconazole, itraconazole, clarithromycin) or the prescriber must verify the dose of Skyclarys™ will be adjusted during concomitant use according to package labeling; AND
Member must not be taking concurrent strong or moderate CYP3A4 inducers (e.g., rifampin, carbamazepine, phenytoin, St. John’s wort, long-acting barbiturates, bosentan, efavirenz, etravirine); AND
Member must not have severe hepatic impairment (Child-Pugh class C); AND
Prescriber must verify liver function tests (LFTs) (e.g., ALT, AST, bilirubin) will be monitored prior to initiation of Skyclarys™ treatment, every month for the first 3 months of treatment, and periodically thereafter or as clinically indicated; AND
Prescriber must verify that B-type natriuretic peptide (BNP) will be assessed prior to initiation of Skyclarys™ and cardiac function will be monitored as clinically indicated; AND
Prescriber must verify lipid parameters will be monitored prior to initiation of Skyclarys™ treatment and periodically thereafter or as clinically indicated; AND
Female members must not be pregnant, must have a negative pregnancy test prior to initiation of therapy, and must agree to use effective non-hormonal contraception during therapy and for 28 days after discontinuation of therapy; AND
Approvals will be for the duration of 1 year. For each subsequent approval, the prescriber must document that the member is responding to the medication, as indicated by slower disease progression and/or other documentation of a positive clinical response to therapy; AND
A quantity limit of 90 capsules per 30 days will apply.

Skysona® (Elivaldogene Autotemcel) Approval Criteria:

An FDA approved diagnosis of early, active cerebral adrenoleukodystrophy (CALD) in male members 4 to 17 years of age; AND
Diagnosis must be confirmed by all of the following:
- Molecular genetic testing confirming a mutation in the ABCD1 gene; AND
  - Members must not have a full deletion of the ABCD1 gene; AND
- Lab results indicating elevated very long-chain fatty acids (VLCFAs); AND
- Active central nervous system (CNS) disease established by central radiographic review of brain magnetic resonance imaging (MRI) demonstrating the following:
  - Loes score between 0.5 and 9 on the 34-point scale; AND
  - Gadolinium enhancement (GdE+) on MRI of demyelinating lesions; AND
- Neurological Function Score (NFS) of ≤1; AND
Skysona^® must be prescribed by a neurologist, endocrinologist, or hematologist/oncologist with expertise in the treatment of CALD and the administration of Skysona^®; AND
Member must not have a known and available human leukocyte antigen (HLA)-matched sibling donor; AND
Member must not have a prior history of hematopoietic stem cell transplantation (HSCT); AND
Member must not be taking statins, Lorenzo’s oil, or dietary regimens used to lower VLCFA levels; AND
Member must not have an immediate family member with known or suspected familial cancer syndrome (FCS); AND
Member must have a negative serology test for human immunodeficiency virus (HIV) prior to apheresis according to the package labeling; AND
Prescriber must verify the member is clinically stable and eligible to undergo HSCT (HSCT must be appropriate for a member to be treated with Skysona^®); AND
Members of reproductive potential must use an effective method of contraception from the start of mobilization through at least 6 months after administration of Skysona^®; AND
Prescriber must verify members of reproductive potential have been counseled on the potential effects of myeloablative conditioning on fertility and the potential risk of infertility is acceptable to the member or member’s caregiver; AND
Prescriber must evaluate the potential for drug interactions, according to package labeling, prior to and after administration of Skysona^®; AND
Prescriber must verify member will be monitored for hematologic malignancies lifelong, with a complete blood count (with differential) performed at month 6 and month 12 after treatment with Skysona^®, then at least annually thereafter for at least 15 years, and with integration site analysis at months 6, 12, and as warranted; AND
Skysona^® must be administered at a Skysona^® qualified treatment center, and the receiving facility must have a mechanism in place to track the patient-specific Skysona^® dose from receipt to storage to administration; AND
Approvals will be for 1 dose per member per lifetime.

Sohonos™ (Palovarotene) Approval Criteria:

An FDA approved diagnosis of fibrodysplasia ossificans progressiva (FOP); and
- Diagnosis must be confirmed by genetic testing identifying a pathogenic R206H mutation in the ACVR1 gene (results of genetic testing must be submitted); and
Member must be:
- 8 years of age or older for female members; or
- 10 years of age or older for male members; and
For members younger than 14 years of age, member’s recent weight (taken within the past 3 weeks) must be provided in order to ensure appropriate dosing in accordance with package labeling; and
Must be prescribed by a geneticist or other specialist with expertise in the treatment of FOP; and
Female members of reproductive potential must not be pregnant and must have a negative pregnancy test within 1 week prior to therapy initiation; and
Prescriber must verify female members of reproductive potential are not breastfeeding and will use effective contraception at least 1 month prior to initiating treatment with Sohonos™ and for 1 month after the last dose of Sohonos™; and
Prescriber must verify the member does not have severe renal impairment (creatinine clearance <30mL/min) or moderate or severe hepatic impairment (Child-Pugh B or C); and
Member must not be taking any of the following medications concomitantly with Sohonos™:
- Strong CYP3A4 inhibitors (e.g., ketoconazole, itraconazole, clarithromycin); or
- Strong or moderate CYP3A4 inducers (e.g., rifampin, carbamazepine, phenytoin, St. John’s wort, phenobarbital, primidone); or
- Vitamin A at doses higher than the recommended daily allowance (RDA); or
- Other oral retinoids (e.g., acitretin, isotretinoin, tretinoin); or
- Tetracyclines (e.g., doxycycline, minocycline, tetracycline); and
If concurrent use with a moderate CYP3A4 inhibitor (e.g., ciprofloxacin, diltiazem, erythromycin, imatinib, fluconazole, fluvoxamine, verapamil) is required, prescriber must agree to reduce the Sohonos™ dose as recommended in the package labeling; and
Prescriber must verify the member or member’s caregiver has been counseled on all warnings and precautions related to Sohonos™, including the risks of embryo-fetal toxicity, premature epiphyseal closure, metabolic bone disorders, psychiatric disorders, and night blindness; and
The request must specify if it is for a chronic daily dose or a flare-up dose; and
Chronic Daily Dose Approvals: Initial approvals will be for the duration of 6 months for the appropriate dose based on member age or weight. For additional approval consideration after 6 months, the prescriber must verify the member is tolerating and responding well to the medication. Subsequent approvals will be for the duration of 1 year; and
Flare-Up Dose Approvals: Initial approvals will be for the duration of 12 weeks for the appropriate doses based on member age or weight. After 12 weeks, flare-up dosing may be approved in additional 4-week increments if the prescriber documents the flare-up symptoms have not resolved at the end of the 12-week period; and
Member will not be approved for the chronic daily dose and flare-up dosing at the same time.

nusinersen (Spinraza™) Approval Criteria:

A diagnosis of spinal muscular atrophy (SMA):
- Type I; OR
- Type II; OR
- Type III with symptoms; AND
Molecular genetic testing to confirm biallelic pathogenic variants in the survival motor neuron gene 1 (SMN1); AND
Member is not currently dependent on permanent continuous ventilation (defined as at least 16 hours of respiratory assistance per day continuously for more than 21 days in the absence of an acute, reversible illness or a perioperative state); AND
Spinraza™ must be prescribed by a neurologist or specialist with expertise in treatment of SMA (or be an advanced care practitioner with a supervising physician who is a neurologist or specialist with expertise in treatment of SMA); AND
Member must not have previously received treatment with Zolgensma® (onasemnogene abeparvovec-xioi); AND
Member will not be approved for concomitant treatment with Evrysdi™ (risdiplam); AND
Platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose and verification that levels are acceptable to the prescriber; AND
Spinraza™ must be administered in a healthcare facility by a specialist experienced in performing lumbar punctures; AND
- Spinraza® must be shipped to the facility where the member is scheduled to receive treatment; AND
A baseline assessment must be provided using at least one of the following exams as functionally appropriate:
- Hammersmith Infant Neurological Exam (HINE); OR
- Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND); OR
- Upper Limb Module (ULM) Test; OR
- Hammersmith Functional Motor Scale Expanded (HFMSE); AND
Initial authorizations will be for the duration of six months, at which time the prescriber must verify the member is responding to the medication as demonstrated by clinically-significant improvement or maintenance of function from pretreatment baseline status using the same exam as performed at baseline assessment:
- Hammersmith Infant Neurological Exam (HINE); OR
- Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND); OR
- Upper Limb Module (ULM) Test; OR
- Hammersmith Functional Motor Scale Expanded (HFMSE); AND
Initial authorizations will be for the duration of six months, at which time the prescriber must verify the member is responding to the medication as demonstrated by clinically-significant improvement or maintenance of function from pretreatment baseline status using the same exam as performed at baseline assessment:
Hammersmith Infant Neurological Exam (HINE); OR
Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND); OR
Upper Limb Module (ULM) Test; OR
Hammersmith Functional Motor Scale Expanded (HFMSE); AND
Approval quantity will be based on Spinraza™ prescribing information and FDA approved dosing regimen(s).
- Only one 5mL vial of Spinraza® is to be dispensed prior to each scheduled procedure for administration.

Prior Authorization form-Spinraza

Tegsedi™ (inotersen) Approval Criteria:

An FDA approved indication for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis; AND
Diagnosis confirmed by the following:
- Tissue (fat pad) biopsy confirming amyloid deposits; OR
- Genetic confirmation of transthyretin (TTR) gene mutation (e.g., Val30Met); AND
Prescriber must verify member is currently experiencing signs and symptoms of polyneuropathy and other causes of polyneuropathy have been ruled out; and
Tegsedi™ must be prescribed by or in consultation with a cardiologist, geneticist, or neurologist or an advanced care practitioner with a supervising physician who is a cardiologist, geneticist, or neurologist; AND
Prescriber must confirm the member will take the recommended daily allowance of vitamin A; AND
Prescriber must agree to monitor ALT, AST, and total bilirubin prior to initiation of Tegsedi™ and every 4 months during treatment; AND
Prescriber must confirm the first injection of Tegsedi™ administered by the patient or caregiver will be performed under the guidance of a health care professional; AND
Prescriber must confirm the patient or caregiver has been trained by a health care professional on the subcutaneuos (sub-Q) administration and proper storage of Tegsedi™; AND
Prescriber must confirm the member has not undergone a liver transplant; AND
Tegsedi™ will not be approved for concomitant use with Amvuttra™ (vutrisiran), Onpattro™, Vyndaqel® (tafamidis meglumine), or Vyndamax™ (tafamidis); AND
Prescriber, pharmacy, and member must be enrolled in the Tegsedi™ Risk Evaluation and Mitigation Strategy (REMS) program and maintain enrollment throughout therapy; AND
Tegsedi™ approvals will be for the duration of 1 year. Reauthorization may be granted if the prescriber documents the member is responding well to treatment and member has not undergone a liver transplant; AND
A quantity limit of four syringes per 28 days will apply.

Prior Authorization form

Veopoz™ (Pozelimab-bbfg) Approval Criteria:

An FDA approved diagnosis of CD55-deficient protein-losing enteropathy (PLE) confirmed by all of the following:
- Genetic testing identifying biallelic pathogenic mutations in the CD55 gene (results of genetic testing must be submitted); and
- A history of PLE; and
Member has active disease defined by hypoalbuminemia (serum albumin concentration ≤3.2g/dL) with 1 or more of the following signs or symptoms within the last 6 months: abdominal pain, diarrhea, peripheral edema, or facial edema; and
Member must be 1 year of age or older; and
Prescriber must verify the member has received the meningococcal vaccine 2 weeks prior to treatment unless urgent treatment is needed; and
Veopoz™ must be prescribed by, or in consultation with, a gastroenterologist, geneticist, hematologist, or other specialist with expertise in the treatment of CD55-deficient PLE; and
The prescriber must verify that Veopoz™ will be administered by a health care professional; and
The member’s recent weight must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to package labeling; and
Initial approvals will be for the duration of 6 months. Further approval may be granted if the prescriber documents that the member is responding well to treatment as indicated by a normalization of serum albumin or documentation of a positive clinical response to therapy.

Vyjuvek™ (Beremagene Geperpavec-svdt) Approval Criteria:

An FDA approved indication for the treatment of wounds in members 6 months of age and older with dystrophic epidermolysis bullosa (DEB); and
Diagnosis must be confirmed by a mutation in the collagen type VII alpha 1 chain (COL7A1) gene (results of genetic testing must be submitted); and
Vyjuvek™ must be prescribed by a dermatologist or other specialist with expertise in the treatment of DEB (or an advanced care practitioner with a supervising physician who is a dermatologist or other specialist with expertise in the treatment of DEB); and
Pharmacy or prescriber must confirm Vyjuvek™ will be prepared by a pharmacist trained in the preparation of Vyjuvek™ prior to administration and must confirm Vyjuvek™ will be shipped to the administering provider via cold chain supply and adhere to the storage and handling requirements in the Vyjuvek™ package labeling; and
Vyjuvek™ must be administered by a health care professional (HCP) trained in the administration of Vyjuvek™. Approvals will not be granted for self-administration. Prior authorization requests must indicate who will administer Vyjuvek™ and in what setting (i.e., treatment facility, HCP office, home health); and
Prescriber must attest that Vyjuvek™ gel will be dosed per package labeling and applied to the same wound(s) until closed before selecting new wound(s) to treat, and that they will prioritize weekly treatment to previously treated wounds if they re-open; and
Prescriber must attest member or caregiver(s) have been counseled on the precautions prior to and during treatment with Vyjuvek™ that are listed in the package labeling, including avoiding direct contact with treated wounds and dressings for 24 hours following administration; and
Female members must not be pregnant and must have a negative pregnancy test immediately prior to therapy initiation. Female members of reproductive potential must be willing to use effective contraception while on therapy; and
A maximum approval quantity of 1 carton (2.5mL) per week or 4 cartons (10mL) per 28 days will apply; and
Initial approvals will be for 3 months. Subsequent approvals will be for 1 year and may be granted if the prescriber documents the member is responding well to treatment as indicated by the presence of wound healing.

Vyndaqel® (Tafamidis Meglumine) and Vyndamax™ (Tafamidis) Approval Criteria:

An FDA approved indication for the treatment of cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular (CV) mortality and CV-related hospitalization; AND
Diagnosis confirmed by:
- Genetic confirmation of transthyretin (TTR) mutation (e.g., Val122Ile) or wild-type amyloidosis; AND
- Cardiac imaging (including ultrasound or MRI) confirming cardiac involvement; AND
Presence of amyloid deposits confirmed by:
- Nuclear scintigraphy; OR
- Endomyocardial biopsy; AND
Member must have medical history of heart failure (NYHA Class I to III); AND
Prescriber must confirm light-chain amyloidosis (AL) has been ruled out; AND
Prescriber must confirm the member has not undergone a liver transplant; AND
Vyndaqel® or Vyndamax™ must be prescribed by or in consultation with a cardiologist or geneticist (or an advanced care practitioner with a supervising physician who is a cardiologist or geneticist); AND
Prescriber must verify Vyndaqel® or Vyndamax™ will not be used concomitantly with Amvuttra™ (vutrisiran), Onpattro® (patisiran) or Tegsedi™ (inotersen); AND
Initial approvals will be for the duration of 6 months. Reauthorization may be granted if the prescriber documents the member is responding well to treatment and member has not undergone a liver transplant; AND
A quantity limit of 4 Vyndaqel® capsules or 1 Vyndamax™ capsule per day will apply.

Prior Authorization form

Zolgensma® (Onasemnogene Abeparvovec-xioi) Approval Criteria:

An FDA approved diagnosis of spinal muscular atrophy (SMA) in pediatric patients younger than 2 years of age; AND
Member must have reached full-term gestational age prior to Zolgensma® infusion; AND
Molecular genetic testing to confirm bi-allelic mutations in the survival motor neuron 1 (SMN1) gene; AND
Member is not currently dependent on permanent invasive ventilation (defined as at least 16 hours of respiratory assistance per day continuously for more than 21 days in the absence of an acute, reversible illness or a perioperative state); AND
Zolgensma® must be prescribed by a neurologist or specialist with expertise in the treatment of SMA (or be an advanced care practitioner with a supervising physician who is a neurologist or specialist with expertise in the treatment of SMA); AND
Member must have baseline anti-AAV9 antibody titers ≤1:50; AND
Prescriber must agree to monitor liver function tests, platelet counts, and troponin-I at baseline and as directed by the Zolgensma® prescribing information; AND
Prescriber must agree to administer systemic corticosteroids starting 1 day prior to the Zolgensma® infusion and continuing as recommended in the prescribing information based on member’s liver function; AND
Zolgensma® must be shipped to the facility where the member is scheduled to receive treatment and must adhere to the storage and handling requirements in the Zolgensma® prescribing information; AND
Member will not be approved for concomitant treatment with Evrysdi™ (risdiplam) or Spinraza^® (nusinersen) following Zolgensma^® infusion (current authorizations for risdiplam or nusinersen will be discontinued upon Zolgensma^® approval); AND
Member’s recent weight must be provided to ensure accurate dosing in accordance with Zolgensma® prescribing information; AND
Only 1 Zolgensma® infusion will be approved per member per lifetime.

Prior Authorization form - Zolgensma

Zokinvy™ (Lonafarnib) Approval Criteria:

An FDA approved indication of 1 of the following:
- To reduce the risk of mortality in Hutchinson-Gilford Progeria Syndrome (HGPS); or
- Treatment of processing-deficient Progeroid Laminopathies (PL) with either:
  - Heterozygous LMNA mutation with progerin-like protein accumulation; or
  - Homozygous or compound heterozygous ZMPSTE24 mutations; and
Member must have confirmatory mutational analysis showing mutation in the LMNA gene; and
Zokinvy™ will not be approved for other progeroid syndromes or processing-proficient PL (based upon its mechanism of action, Zokinvy™ would not be effective in these populations); and
Member must be 1 year of age or older; and
Member must have a body surface area (BSA) ≥0.39m²; and
Member must have clinical signs of progeria (e.g., characteristic facial features, growth deficiency, atherosclerosis); and
Zokinvy™ must be prescribed by, or in consultation with, a specialist with expertise in treating HGPS or PL (or an advanced care practitioner with a supervising physician who is a specialist in treating HGPS or PL); and
Member must not be taking any of the following medications: strong/moderate CYP3A inhibitors, CYP2C9 inhibitors, midazolam, lovastatin, simvastatin, atorvastatin, or loperamide if younger than 2 years of age; and
Prior to and during treatment, the potential for drug interactions should be considered, concomitant medications reviewed, and members should be monitored for adverse reactions; and
Member should have ophthalmological evaluations performed at regular intervals and at the onset of any new visual changes; and
Prescriber must verify the member will be monitored for changes in electrolytes, complete blood counts, renal function, and liver enzymes; and
Member’s recent BSA must be provided on the prior authorization request in order to authorize the appropriate amount of drug required according to the package labeling; and
The maximum approvable dose of Zokinvy™ is 300mg/m² per day; and
Initial approvals will be for 6 months. After 6 months of utilization, compliance and information regarding efficacy, such as a positive response to treatment including no new or worsening heart failure and no stroke incidence, will be required for continued approval. Subsequent approvals will be for 12 months and compliance and documentation of a positive response to Zokinvy™ therapy will be required on each continuation request.

Zynteglo^® (Betibeglogene Autotemcel) Approval Criteria:

An FDA approved indication for the treatment of adult and pediatric members with beta thalassemia who require regular red blood cell (RBC) transfusions; AND
Member must be 4 years of age or older; AND
Member must weigh ≥6kg; AND
Member must require regular RBC transfusions as demonstrated by the following:
- History of ≥100mL/kg/year transfusions of packed RBCs in the last 2 years; OR
- ≥8 transfusions of packed RBCs per year in the last 2 years; AND
Zynteglo^® must be prescribed by a hematologist with expertise in the treatment of beta thalassemia and the administration of Zynteglo^®; AND
Member must not have a known and available human leukocyte antigen (HLA)-matched sibling donor; AND
Member must not have a prior history of hematopoietic stem cell transplantation (HSCT); AND
Member must have a negative serology test for human immunodeficiency virus (HIV) prior to apheresis; AND
Prescriber must verify the member is clinically stable and eligible to undergo HSCT (HSCT must be appropriate for a member to be treated with Zynteglo^®); AND
Female members must not be pregnant and must have a negative pregnancy test prior to the start of mobilization, prior to conditioning procedures, and prior to Zynteglo^® administration; AND
Male and female members of reproductive potential must use an effective method of contraception from the start of mobilization through at least 6 months after administration of Zynteglo^®; AND
Prescriber must verify male and female members of reproductive potential have been counseled on the potential effects of myeloablative conditioning on fertility and the potential risk of infertility is acceptable to the member; AND
Prescriber must evaluate the potential for drug interactions, according to package labeling, prior to and after administration of Zynteglo^®; AND
Member will not be approved for treatment with Reblozyl^® (luspatercept-aamt) following Zynteglo^® infusion (current authorizations for luspatercept-aamt will be discontinued upon Zynteglo^® approval); AND
Prescriber must verify member will be monitored for hematologic malignancies lifelong, with a complete blood count (with differential) performed at month 6 and month 12 after treatment with Zynteglo^®, then at least annually thereafter for at least 15 years, and with integration site analysis at months 6, 12, and as warranted; AND
Zynteglo^® must be administered at a Zynteglo^® qualified treatment center, and the receiving facility must have a mechanism in place to track the patient-specific Zynteglo^® dose from receipt to storage to administration; AND
Approvals will be for 1 dose per member per lifetime.

Last Modified on Apr 19, 2024

Genetic Disorders

Adakveo®

Alpha1-Proteinase Inhibitors

Amvuttra™ / Onpattro™

Bylvay™

Carbaglu®

Crysvita®

Cuvrior™

Daybue™

Dojolvi®

Duchenne muscular dystrophy

Emflaza®

Endari™

Enjaymo™

Evrysdi™

Firdapse®

Gamifant®

Imcivree™

Jesduvroq™

Joenja®

Jynarque™

Livmarli™

Luxturna™

Lysosomal Storage Disease

Nulibry™

Oxbryta®

Oxlumo™

Pyrukynd®

Reblozyl®

Revcovi™

Ruzurgi®

Ryplazim®

Siklos®

Skyclarys™

Skysona®

Sohonos™

Spinraza™

Tegsedi™

Veopoz™

Vyjuvek™

Vyndagel®/Vyndamax™

Zolgensma®

Zokinvy™

Zynteglo®