Kuvan (sapropterin dihydrochloride) — Blue Cross Blue Shield of Montana

Initial criteria

• 1. Patient has a diagnosis of phenylketonuria (PKU) AND
• 2. If the patient has an FDA labeled indication, then ONE of the following: (A) Patient’s age is within FDA labeling for the requested indication and agent OR (B) Support exists for using the requested agent for the patient’s age for the requested indication AND
• 3. ONE of the following: (A) BOTH of the following: (1) Phenylalanine levels cannot be maintained within recommended maintenance range with dietary intervention despite strict compliance AND (2) Phe-restricted diet will continue while being treated OR (B) If Palynziq is requested, current phenylalanine level < 360 micromol/L (6 mg/dL) AND
• 4. If requested agent is Kuvan or sapropterin, then ONE of the following: (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 blood Phe > 360 micromol/L (6 mg/dL) AND
• 5. If requested agent is Palynziq, patient has baseline blood Phe > 600 micromol/L (10 mg/dL) AND
• 6. If request is for a brand agent, then ONE of the following: (A) Patient currently stable on requested agent OR (B) Tried and inadequate response to generic sapropterin OR (C) Intolerance/hypersensitivity to generic sapropterin OR (D) Contraindication to generic sapropterin OR (E) Generic sapropterin discontinued due to lack of efficacy/adverse event OR (F) Generic sapropterin expected to be ineffective or cause significant issues OR (G) Generic sapropterin not in best interest of patient OR (H) Patient tried another drug in same class with lack of efficacy/adverse event OR (I) Support for brand agent use (e.g., two null mutations in trans) AND
• 7. Prescriber is a specialist in metabolic disorders or has consulted one AND
• 8. Patient will NOT use with another targeted agent in this program AND
• 9. Patient does not have any FDA-labeled contraindications to the requested agent AND
• 10. Requested dose within FDA labeling

Reauthorization criteria

• 1. Patient previously approved for requested agent through plan’s PA process AND
• 2. Patient has improvements or stabilization as indicated by ONE of the following: (A) If Kuvan or sapropterin then ONE of: (1) Blood Phe maintained within acceptable range [<12 years or pregnant: 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) If Palynziq then ONE of: (1) Blood Phe ≤ 600 micromol/L (10 mg/dL) OR (2) ≥20% decrease in blood Phe from baseline OR (3) Has not received 16 weeks of therapy at maximum labeled dose and prescriber will evaluate for dose escalation AND
• 3. ONE of the following: (A) Patient continues Phe-restricted diet OR (B) If Palynziq requested, phenylalanine level < 360 micromol/L (6 mg/dL) AND
• 4. If request is for brand agent, ONE of: (A) Patient stable on requested agent OR (B) Tried and inadequate response to generic sapropterin OR (C) Intolerance/hypersensitivity to generic sapropterin OR (D) Contraindication to generic sapropterin OR (E) Generic sapropterin discontinued due to lack of efficacy/adverse events OR (F) Generic sapropterin expected to be ineffective/problematic OR (G) Generic sapropterin not in patient’s best interest OR (H) Tried another drug in same class with inadequate response/adverse event OR (I) Support for brand agent use (e.g., two null mutations in trans) AND
• 5. Prescriber is specialist in metabolic disorders or consulted one AND
• 6. Patient will NOT use with another targeted agent included in this program AND
• 7. No FDA-labeled contraindications AND
• 8. Dose within labeled dosing

Approval duration

Kuvan 3 months (BCBSMT), Palynziq 9 months (BCBSMT), Sephience 3 months (BCBSMT); otherwise Kuvan 1–2 months (depending on dose), Palynziq 9 months, Sephience 1–2 months (depending on dose); renewal 12 months