- 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.
Genetic Disorders
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.
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.
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.
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.
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.
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.
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.
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.
Evrysdi™ (Risdiplam) Approval Criteria:
- An FDA approved diagnosis of spinal muscular atrophy (SMA) in members 2 months of age and older; 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 monitor member’s liver function prior to initiating Evrysdi™ and periodically while receiving Evrysdi™ treatment; 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.
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.
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®.
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).
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 proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency; and
- Molecular genetic testing to confirm variants in the POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance; 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, obesity associated with other genetic syndromes, or general obesity will not be approved; and
- Member’s baseline weight and body mass index (BMI) must be provided; and
- Baseline BMI must be ≥30kg/m2 for adults or ≥95th 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 moderate, severe, or end stage renal disease [estimated glomerular filtration rate (eGFR) <60mL/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
- 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; and
- A quantity limit of 9mL 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.
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.
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
- 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.
An FDA approved indication for the treatment of sickle cell disease (SCD) in members 12 years of age and older; AND
Member must have a history of vaso-occlusive crises (VOCs); AND
Member must have baseline hemoglobin ≥5.5 to ≤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
The member must not be taking concomitant strong CYP3A4 inhibitors (e.g., fluconazole, ketoconazole) or the prescriber must verify the dose of Oxbryta® will be reduced during concomitant use according to package labeling; 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
A quantity limit of 3 tablets per day will apply; 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.
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
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 for the 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; AND
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.
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®.
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.
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.
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.
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; 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.
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.
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.
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.
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.
Last Modified on
Mar 29, 2021
