Metabolic Disorders

colic acid (Cholbam™) Approval Criteria:

• 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.

cysteamine bitartrate (Procysbi™) Approval Criteria:

• 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).

glycerol phenylbutyrate (Ravicti®) Approval Criteria:

• 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).

metreleptin (Myalept™) Approval Criteria:

• 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.