Genetic Disorders

 

eliglustat (Cerdelga®)

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   

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imiglucerase (Cerezyme®), taliglucerase alfa (Elelyso®), and velaglucerase alfa (Vpriv®)
imiglucerase (Cerezyme®), taliglucerase alfa (Elelyso®), and velaglucerase alfa (Vpriv®) Approval Criteria:
  • 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   

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miglustat (Zavesca®)

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  

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eteplirsen (Exondys 51™)

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  

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nusinersen (Spinraza™)

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
  • 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 

 Prior Authorization form-Spinraza  

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Alpha1-Proteinase Inhibitor [Human]

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.

 

Aralast NP™ and Glassia® (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.

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, Aralast NP™, or Glassia®; 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  

       

l-glutamine (Endari™)

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  

       

eculizumab (Soliris®)

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