asfotase alfa (Strensiq™) Approval Criteria:
- 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.
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).
dichlorphenamide (Keveyis™) Approval Criteria:
- 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.
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.
Prior Authorization form