Genetic Disorders

eliglustat (Cerdelga®) Approval Criteria:

• An FDA approved indication of Type 1 Gaucher disease (GD1); AND
• Member is classified as one 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
• Prescriber must verify that the member will not take Cerdelga® concurrently with another therapy for GD1. 
• 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. 
• Approvals will be for the duration of six months, at which time the prescriber must verify the patient is responding to the medication.  

Prior Authorization form   

• A diagnosis of symptomatic (e.g., anemia, thrombocytopenia, bone disease, splenomegaly, or hepatomegaly) Type 1 or Type 3 Gaucher disease (GD); AND
• Member’s weight (kg) must be provided and have been taken within the last four weeks to ensure accurate weight based dosing; AND
• Prescriber must verify that the member will not take requested therapy concurrently with another therapy for GD. 
• Approvals will be for the duration of six months, at which time the prescriber must verify the patient is responding to the medication.

Prior Authorization form   

miglustat (Zavesca®) Approval Criteria:  

• An FDA approved indication of mild/moderate Type 1 Gaucher disease (GD1); AND
• A patient-specific, clinically significant reason why the member cannot use one of the following enzyme replacement therapies: 
• Cerezyme® (imiglucerase); OR
• Elelyso® (taliglucerase alfa); OR
• Vpriv® (velaglucerase alfa); AND
• Prescriber must verify that the member will not take Zavesca® concurrently with another therapy for GD1. 
• A quantity limit of 90 capsules per 30 days will apply.   
• Approvals will be for the duration of six months, at which time the prescriber must verify the patient is responding to the medication.  

 Prior Authorization form  

eteplirsen (Exondys 51™) Approval Criteria:

• An FDA approved diagnosis of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping; 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  

 deflazacort (Emflaza®) Approval Criteria:

• An FDA approved diagnosis of Duchenne muscular dystrophy (DMD); AND  
• Member must be 5 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  

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   

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; 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
• 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  

Crysvita® (Burosumab-twza) Approval Criteria:  

• An FDA approved indication for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year 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’s XLH symptoms must not be adequately controlled on phosphate and calcitriol supplements. Members experiencing adverse effects related to these treatments may also be considered for approval. Detailed information regarding adverse effects must be documented on the prior authorization request; 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
   
• 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.

Prior Authorization form   

Prolastin®-C (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.

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
• A patient-specific, clinically significant reason why the member cannot use Prolastin®-C, Aralast NP™, Glassia®, and Zemaira®; 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  

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
• A patient-specific, clinically significant reason why NutreStore® (L-glutamine powder for oral solution) cannot be used must be provided; 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  

NutreStore® (L-Glutamine) Approval Criteria [Short Bowel Syndrome Diagnosis]:

• An FDA approved diagnosis of Short Bowel Syndrome; AND
• NutreStore® must be used in conjunction with a recombinant human growth hormone that is approved for this indication; AND
• Member must be receiving optimal management of Short Bowel Syndrome (e.g., specialized oral diet, enteral feedings, parenteral nutrition, fluid and micronutrient supplements); AND  
• Approvals will be for up to 16 weeks.

NutreStore® (L-Glutamine) Approval Criteria [Sickle Cell Disease Diagnosis]:

• A 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
• NutreStore® 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. 
• 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  

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  
• Member must not be given live vaccines while on Siklos® 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   

eculizumab (Soliris®) Approval Criteria [Generalized Myasthenia Gravis (gMG) Diagnosis]:

• An FDA approved diagnosis of gMG; AND
• Positive serologic test for anti-acetylcholine receptor (AchR) antibodies; AND
• Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IV; a AND
• Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score ≥6; AND
• Member must meet one of the following:
  
  • Failed treatment over one year or more with two or more immunosuppressive therapies (ISTs) either in combination or as monotherapy; OR  
  • Failed at least one IST and required chronic plasmapheresis or plasma exchange (PE) or intravenous immunoglobulin (IVIG); AND
   
• Initial approvals will be for the duration of six months at which time an updated MG-ADL score must be provided. Continued authorization requires improvement in the MG-ADL score from baseline. Subsequent approvals will be for the duration of one year.  

 Prior Authorization form  

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