Metabolic Disorders

PA Approval Criteria:

• An FDA-approved diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) also known as tripeptidyl peptidase-1 (TPP-1) deficiency; AND
• Member must have confirmed TPP-1 enzymatic deficiency via enzyme assay, confirmed by molecular analysis; AND
• Member must be at least 3 years of age or older; AND
• Brineura™ must be prescribed by a specialist with expertise in treatment of CLN2 (or be an advanced care practitioner with a supervising physician who is a specialist with expertise in treating CLN2); AND
• Brineura™ must be administered in a healthcare facility by a prescriber who is knowledgeable in intraventricular administration; AND
• Member must not have ventriculoperitoneal shunts or acute intraventricular access device-related complications; AND
• Member must not have documented generalized status epilepticus within 4 weeks of initiating treatment; AND
• Prescriber must verify member’s blood pressure and heart rate will be monitored prior to each infusion, during infusion, and post-infusion; AND
• Prescriber must be willing to perform regular 12-lead electrocardiogram (ECG) evaluation at baseline and at least every 6 months and verify that they are acceptable to the prescriber; AND
• A baseline assessment must be performed to assess the Motor plus Language CLN2 score; AND
• Initial authorizations will be for the duration of six months, at which time compliance will be required for continued approval. After 12 months of utilization, the prescriber must verify the member is responding to the medication as demonstrated by a two point or less decline in Motor plus Language CLN2 score from baseline; AND
• Approval quantity will be based on Brineura™ prescribing information and FDA approved dosing regimen. 

• An FDA approved diagnosis of one of the following:
  • Treatment of bile acid disorders due to single enzyme defects (SEDs); OR
  • Adjunctive treatment of peroxisomal disorders (PDs) including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption; AND   
• Treatment with Cholbam™ should be initiated and monitored by a hepatologist or pediatric gastroenterologist; AND
• The prescriber must verify that AST, ALT, GGT, alkaline phosphatase, bilirubin and INR will be monitored every month for the first three months, every three months for the next nine months, every six months during the next three years and annually thereafter; AND
• Cholbam™ should be discontinued if liver function does not improve within three months of starting treatment, if complete biliary obstruction develops, or if there are persistent clinical or laboratory indicators of worsening liver function or cholestasis; AND
• Initial approvals will be for the duration of three months to monitor for compliance and liver function tests.
• Continuation approvals will be granted for the duration of one year. 
• A quantity limit of 120 capsules per 30 days will apply.  Quantity limit requests will be based on the member’s recent weight taken within the last 30 days.

PA criteria:

• An FDA approved diagnosis of Hunter syndrome (mucopolysaccharidosis type II; MPS II) confirmed by:
  • Enzyme assay demonstrating a deficiency of iduronate-2-sulfatase enzyme activity; OR
  • Molecular genetic testing confirming a hemizygous pathogenic variant in the IDS gene; 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. 

agalsidase beta (Fabrazyme®) Approval Criteria:

• An FDA approved diagnosis of Fabry disease. Diagnosis must be confirmed by one of the following:
  • Genetic testing confirming positive galactosidase alpha (GLA) gene mutation; OR
  • Decreased plasma levels of alpha-galactosidase A (less than 5% of normal); AND
• Fabrazyme® (agalsidase beta) will initially be approved for six months. After that time, compliance will be required for continued authorization; 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.    

PA Approval Criteria:

• An FDA approved diagnosis of Fabry disease with a confirmed amenable GLA gene variant based on in vitro assay data; AND
• Galafold™ must be prescribed in consultation with a geneticist or an advanced care practitioner with a supervising physician who is a geneticist; AND  
• Member must have an estimated glomerular filtration rate (eGFR) of at least 30mL/min/1.73m2; AND
• Galafold™ will not be approved for concomitant use with enzyme replacement therapy (ERT); AND  
• Galafold™ will initially be approved for six months. After that time, compliance will be required for continued authorization; AND
• A quantity limit of 14 capsules per 28 days will apply.

PA Approval Criteria:

• An FDA approved diagnosis of acute hepatic porphyria (AHP) confirmed by:
  
  • Genetic testing; OR
  • Elevated urinary porphobilinogen (PBG) and signs/symptoms of AHP; AND
   
• Member must be 18 years of age or older; AND  
• Givlaari™ must be administered in a health care setting by a health care professional prepared to manage anaphylaxis; AND
  
  • Givlaari™ must be shipped to the health care setting where the member is scheduled to receive treatment; AND
   
• The prescriber must agree to monitor liver function tests prior to initiating treatment with Givlaari™, every month during the first 6 months of treatment, and as clinically indicated thereafter; AND  
• The prescriber must agree to monitor renal function during treatment with Givlaari™ as clinically indicated; AND
• Member must not be taking sensitive CYP1A2 or CYP2D6 substrates (e.g., caffeine, dextromethorphan, duloxetine, amitriptyline, olanzapine, fluoxetine, paroxetine, hydrocodone, tramadol) concomitantly with Givlaari™; 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. Further approval may be granted if the prescriber documents that the member is responding well to treatment as indicated by less porphyria attacks and that the member does not have elevated transaminase levels.

sebelipase alfa (Kanuma®) Approval Criteria:

• An FDA approved diagnosis of Lysosomal Acid Lipase (LAL) deficiency; AND
• Kanuma® (sebelipase alfa) must be administered in a healthcare setting by a healthcare professional prepared to manage anaphylaxis; 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.

• An FDA approved indication for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, or related variants; AND 
• Prescriber documentation that all non-pharmacological treatments failed including the following:
  • Hyperkalemic periodic paralysis:
    • Acute attacks can be aborted with sugar or mild exercise 
    • Avoiding foods rich in potassium 
    • Avoiding fasting 
    • High-carbohydrate diet 
    • Avoiding strenuous activity 
    • Avoiding prolonged cold exposure 
  • Hypokalemic periodic paralysis:
    • Low-carbohydrate diet (avoiding carbohydrate loading) 
    • Avoiding vigorous exercise (some mild attacks can be aborted by low level exercise) 
• Prescriber documentation of frequent and severe attacks requiring pharmacological treatment (at least one attack per week but no more than three attacks per day); AND 
• A four-week trial within the last 90 days of acetazolamide in combination with
  • Spironolactone or triamterene in hypokalemic periodic paralysis; OR 
  • Hydrochlorothiazide in hyperkalemic periodic paralysis 
• A quantity limit of four tablets per day will apply. 
• Initial approvals will be for the duration of three months after which time compliance will be required for continued approval. Additionally, for continuation the prescriber must include information regarding reduced frequency or severity of attacks.

PA criteria:

• An FDA approved diagnosis of phenylketonuria; AND
• Documentation of active management with a phenylalanine restricted diet; AND
• Member must not have two null mutations in trans; AND
• Baseline phenylalanine concentration must be documented on the prior authorization request and must be drawn within the last 30 days; AND
• Concomitant use with Palynziq™ (pegvaliase-pqpz) will not be approved; AND
• Initial approvals will be for the duration of 30 days.  After which time, the prescriber must verify that the member responded to treatment as defined by laboratory documentation of greater than or equal to a 30% decrease in blood phenylalanine levels from baseline.
  • If the member was initiated at 10mg/kg/day dose, then a subsequent trial of 20mg/kg/day for a duration of 30 days can be approved.  After which time, the prescriber must verify that the member responded to treatment as defined by laboratory documentation of greater than or equal to a 30% decrease in blood phenylalanine levels from baseline.
  • If the member was initiated at 20mg/kg/day dose, then no additional approvals will be granted after a trial period of 30 days if the member did not respond to treatment as defined by laboratory documentation of greater than or equal to a 30% decrease in blood phenylalanine levels from baseline. 
• Subsequent approvals will be for the duration of one year. 
• Reauthorization will require the following:
  
  • Documentation of active management with a phenylalanine restricted diet; AND
  • Verification from the prescriber of continued response to therapy. 

Lumizyme® (alglucosidase Alfa) Infantile-Onset Approval Criteria:

• An FDA approved diagnosis of infantile-onset Pompe disease (acid alpha-glucosidase [GAA] deficiency); AND
• Documentation of diagnosis confirmation of GAA enzyme deficiency through specific genetic laboratory test(s); AND
• Lumizyme® must be prescribed by a geneticist or a physician that specializes in the treatment of Pompe disease and/or inherited genetic disorders; AND
• Member’s weight must be provided and have been taken within the last four weeks to ensure accurate dosing.

Lumizyme® (alglucosidase Alfa) Late-Onset (Non-Infantile) Approval Criteria:

• An FDA approved diagnosis of late-onset (non-infantile) Pompe disease (acid alpha-glucosidase [GAA] deficiency); AND
• Documentation of diagnosis confirmation of GAA enzyme deficiency through specific genetic laboratory test(s); AND
• Provider must document presence of symptoms of Pompe disease; AND
• Lumizyme® must be prescribed by a geneticist or a physician that specializes in the treatment of Pompe disease and/or inherited genetic disorders; AND
• Member’s weight must be provided and have been taken within the last four weeks to ensure accurate dosing.  
• Initial approval will be for the duration of six months, at that time compliance and information regarding efficacy, such as improvement or stabilization in Forced Vital Capacity (FVC) and/or 6-minute walk test (6MWT), will be required for continued approval.  Additional authorizations will be for the duration of one year. 

• An FDA approved diagnosis of leptin deficiency in patients with congenital or acquired generalized lipodystrophy; and
• Approvals will not be granted for the following diagnoses:
  • Metabolic disease without current evidence of generalized lipodystrophy
  • HIV-related lipodystrophy
  • General obesity not associated with congenital leptin deficiency
• Myalept™ must be prescribed by an endocrinologist; and
• Prescriber must agree to test for neutralizing antibodies in patients who experience severe infections or if they suspect Myalept™ is no longer effective.
  • Baseline HbA1c, fasting glucose, and fasting triglycerides must be stated on prior authorization request
  • Re-approvals will require recent lab values (HbA1c, fasting glucose, and fasting triglycerides) to ensure neutralizing antibodies have not developed; and
• Prescriber and pharmacy must be enrolled in the Myalept™ REMS program; and
• Approvals will be for the duration of three months to evaluate compliance and ensure the prescriber is assessing continued efficacy; and
• A quantity limit of one vial per day will apply.  

PA Approval Criteria:

• An FDA approved diagnosis to reduce blood phenylalanine concentrations in patients with phenylketonuria who have uncontrolled blood phenylalanine concentrations >600µmol/L on existing management; AND
• Documentation of active management with a phenylalanine restricted diet; AND
• Baseline phenylalanine concentration must be documented on the prior authorization request and must be drawn within the last 30 days; AND
• Documentation the member’s average blood phenylalanine concentration over the last 6 months is >600µmol/L on existing management; AND
• Concomitant use with Kuvan® (sapropterin) will not be approved; AND
• Prescriber, pharmacy, and member must be enrolled in the Palynziq™ Risk Evaluation and Mitigation Strategy (REMS) program and maintain enrollment throughout therapy; AND  
• Initial dose must be administered under the supervision of a health care provider equipped to manage anaphylaxis and observe the member for at least 60 minutes following injection; AND
• Member must be prescribed auto-injectable epinephrine and be counseled on its appropriate use; AND
• Initial approvals will be for the duration of 33 weeks to allow for initial titration and for 24 weeks of maintenance treatment with 20mg once daily dosing.  Patients should then be assessed for a 20% reduction in blood phenylalanine concentration from pre-treatment baseline or a blood phenylalanine concentration ≤600µmol/L.
  
  • If member has not achieved a 20% reduction in blood phenylalanine concentration from pre-treatment baseline or a blood phenylalanine concentration ≤600µmol/L, approvals may be granted for the 40mg once daily dosing for a duration of 16 weeks; OR
  • If member has achieved a 20% reduction in blood phenylalanine concentration from pre-treatment baseline or a blood phenylalanine concentration ≤600µmol/L, subsequent approvals will be for the duration of one year; AND
   
• Members who do not achieve at least a 20% reduction in blood phenylalanine concentration from pre-treatment baseline or a blood phenylalanine concentration ≤600µmol/L after 16 weeks of continuous treatment with the maximum dosage of 40mg once daily will not be approved for subsequent approvals; AND
• Subsequent approvals will be for the duration of one year.
• Reauthorization will require the following:
  • Documentation of active management with a phenylalanine restricted diet; AND
  • Verification from the prescriber of continued response to therapy. 

• An FDA approved diagnosis of nephropathic cystinosis; AND
• A patient specific, clinically significant reason why member cannot use the short-acting formulation Cystagon® (cysteamine bitartrate) must be provided; AND
• Use of Procysbi® granules will require a patient-specific, clinically significant reason why the member cannot use the capsule formulation of Procysbi®.

• An FDA approved diagnosis of urea cycle disorder (UCD); AND
• Active management with protein restricted diet; AND
• A patient specific, clinically significant reason why member cannot use Buphenyl® (sodium phenylbutyrate).

PA Approval Criteria:

• An FDA approved indication to increase pain-free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP); AND  
  • The diagnosis of EPP must be confirmed by genetic testing; AND  
• Member must be 18 years of age or older; AND  
• Scenesse® must be administered by a health care professional who is proficient in the subcutaneous (subQ) implantation procedure and has completed the training program provided by the manufacturer prior to administration of the Scenesse® implant; AND
  • Scenesse® must be shipped via cold chain supply shipping and delivery to the health care setting where the member is scheduled to receive the implant administration; AND
  • Scenesse® must be stored under refrigeration (36 to 46°F) and protected from light prior to implantation; AND  
• The Scenesse® implant should be inserted using an SFM Implantation Cannula or other implantation device that has been determined by the manufacturer to be suitable for implantation of Scenesse®; AND
• The prescriber must agree that the member will be monitored by a health care provider for at least 30 minutes after the implant administration; AND  
• The prescriber must agree that the member will have a full body skin examination performed at least twice yearly while the member is being treated with Scenesse® to monitor pre-existing and new skin pigmentary lesions; AND
• Documentation that member will maintain sun and light protection measures during treatment with Scenesse® to prevent phototoxic reactions related to EPP; AND
• A quantity limit of 1 implant per 60 days will apply. Initial approvals will be for 2 implants for the duration of 4 months. Further approval may be granted if the prescriber documents that the member is responding well to treatment as indicated by increased tolerance of sunlight (i.e., less phototoxic reactions).   

• An FDA approved indication for the treatment of patients with perinatal/infantile-onset and juvenile-onset hypophosphatasia (HPP); AND
• Confirmed diagnosis by laboratory testing of:
  • Low age-adjusted ALP activity; AND
  • Elevated pyridoxal 5’-phophate (PLP) levels; AND 
• Member’s weight (kg) must be provided and have been taken within the last four weeks to ensure accurate weight-based dosing; AND
• The 80mg/0.8mL vial should not be used in pediatric patients weighing less than 40kg.

elosulfase alfa (Vimizim®) Approval Criteria:

• An FDA approved diagnosis of Morquio A syndrome (mucopolysaccharidosis type IVA; MPS IVA) confirmed by:
  • Enzyme assay demonstrating a deficiency of N-acetylgalactosamine-6-sulfatase (GALNS) enzyme activity; OR
  • Molecular genetic testing to confirm biallelic pathogenic variants in GALNS; AND  
• Vimizim® must be administered by a healthcare professional prepared to manage anaphylaxis; AND
• Initial approvals will be for the duration of twelve months. Reauthorization may be granted if the prescriber documents the member is responding well to treatment.
• 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.

Xuriden™ (Uridine Triacetate) Approval Criteria:

• An FDA approved diagnosis of hereditary orotic aciduria defined by at least one of the following:  
  
  • Assay of the orotate phosphoribosyltransferase and orotidylic acid decarboxylase enzymes in the patients erythrocytes showing deficiency in both enzymes or deficiency in orotidylic acid decarboxylase alone; OR
  • Evidence of megaloblastic anemia:     
    Normal serum folate and vitamin B12 levels and no evidence of Transcobalamine II deficiency; OR
  • Orotic acid crystals visualized in the urine via microscopy; AND 
• Current weight of member must be provided on the prior authorization request; AND
  
  • Weights should be reassessed every six months to ensure proper dosing and effectiveness; OR
  • Prescriber can indicate urine orotic acid levels are within normal ranges and dosing remains appropriate; AND 
• The prescriber must verify that the patient/caregiver is able to properly measure and administer medication; AND
• A quantity limit of four packets per day will apply.