Genetic Disorders 2017 Archive

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  

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  

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  

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