Kuvan — Blue Cross Blue Shield of Kansas

Initial criteria

• 1. Patient has a diagnosis of phenylketonuria (PKU)
• 2. If patient has an FDA labeled indication, then ONE of: (A) patient's age is within FDA labeling for requested indication OR (B) there is support for using the requested agent for the patient's age for the requested indication
• 3. ONE of: (A) BOTH: (1) Phenylalanine levels cannot be maintained within the recommended maintenance range with dietary intervention despite strict compliance AND (2) the Phe-restricted diet will continue while treated with the requested agent OR (B) if requested agent is Palynziq, current phenylalanine level is less than 360 micromol/L (6 mg/dL)
• 4. If requested agent is Kuvan or sapropterin, ONE of: (A) patient age < 12 years AND baseline blood Phe > 360 micromol/L (6 mg/dL) OR (B) patient age ≥ 12 years AND baseline blood Phe > 600 micromol/L (10 mg/dL) OR (C) patient planning pregnancy or currently pregnant AND baseline Phe > 360 micromol/L (6 mg/dL)
• 5. If requested agent is Palynziq, baseline blood Phe > 600 micromol/L (10 mg/dL)
• 6. If request is for a brand agent, ONE of: (A) tried and inadequate response to generic sapropterin despite monitored adherence OR (B) intolerance or hypersensitivity to generic sapropterin not expected with brand OR (C) FDA labeled contraindication to generic sapropterin not expected with brand OR (D) support for use of the requested brand agent over generic sapropterin (e.g., presence of two null mutations in trans)
• 7. Prescriber is a specialist in metabolic disorders or has consulted with a specialist
• 8. Patient will NOT be using the requested agent in combination with another targeted agent in this program
• 9. Patient does NOT have any FDA labeled contraindications to the requested agent
• 10. Requested dose within FDA labeled dosing

Reauthorization criteria

• 1. Patient previously approved for the requested agent through plan’s Prior Authorization process
• 2. Patient shows improvement or stabilization as indicated by ONE of: (A) For Kuvan/sapropterin: (1) Blood Phe maintained within acceptable range: <12 years and for females currently pregnant or planning pregnancy: 120-360 micromol/L (2-6 mg/dL); ≥12 years: 120-600 micromol/L (2-10 mg/dL) OR (2) ≥30% decrease in blood Phe from baseline OR (B) For Palynziq: patient shows improvement per program continuation requirements

Approval duration

Kuvan: 1–2 months depending on dose; Palynziq: 9 months; Sephience: 1–2 months depending on dose