Genetic Disorders

Carbaglu® (Carglumic Acid) Approval Criteria:

• An FDA approved diagnosis of N-acetylglutamate synthase (NAGS) deficiency; AND
• Carbaglu® must be prescribed by, or in consultation with, a geneticist; AND
• 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.  

Prior Authorization form   

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 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  

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   

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   

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
• 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  

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 nusinersen following Zolgensma® infusion (current authorizations for 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

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 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. 

Prior Authorization form   

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  

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  

• 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 
• 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 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
• Onpattro™ will not be approved for concomitant use with Tegsedi™,Vyndaqel® (tafamidis meglumine), or Vyndamax™ (tafamidis); 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
• Onpattro™ approvals will be for the duration of 6 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  
• 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  

• 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; AND
  • Genetic confirmation of transthyretin (TTR) gene mutation (e.g., Val30Met); 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  
• Tegsedi™ will not be approved for concomitant use with 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 6 months. Reauthorization may be granted if the prescriber documents the member is responding well to treatment; AND
• A quantity limit of four syringes per 28 days will apply.  

 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  

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

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  

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  
• 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 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  
• A quantity limit of 4 Vyndaqel® capsules or 1 Vyndamax™ capsule per day will apply.

Prior Authorization form