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Palynziq — Blue Cross Blue Shield of Illinois

phenylketonuria (PKU)

Initial criteria

  • The patient has a diagnosis of phenylketonuria (PKU) AND
  • If the patient has an FDA labeled indication, then ONE of the following: (A) The patient’s age is within FDA labeling for the requested indication for the requested agent OR (B) There is support for using the requested agent for the patient’s age for the requested indication AND
  • ONE of the following: (A) BOTH of the following: (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 being treated with the requested agent OR (B) If the requested agent is Palynziq, the patient’s current phenylalanine level is less than 360 micromol/L (6 mg/dL) AND
  • If the requested agent is Kuvan or sapropterin, then ONE of the following: (A) The patient is age < 12 years AND has a baseline blood Phe level > 360 micromol/L (6 mg/dL) OR (B) The patient is age ≥ 12 years AND has a baseline blood Phe level > 600 micromol/L (10 mg/dL) OR (C) The patient is planning on becoming pregnant or is currently pregnant AND has a baseline Phe level > 360 micromol/L (6 mg/dL) AND
  • If the requested agent is Palynziq, the patient has a baseline blood Phe level > 600 micromol/L (10 mg/dL) AND
  • If the request is for a brand agent, then ONE of the following: (A) The patient is currently being treated with and stable on the requested agent OR (B) The patient has tried and had an inadequate response to generic sapropterin OR (C) The patient has an intolerance or hypersensitivity to generic sapropterin that is not expected to occur with the brand OR (D) The patient has an FDA labeled contraindication to generic sapropterin not expected to occur with the brand OR (E) Generic sapropterin was discontinued due to lack of efficacy, diminished effect, or adverse event OR (F) Generic sapropterin is expected to be ineffective based on clinical characteristics or would cause barriers to adherence or harm OR (G) Generic sapropterin is not in the best interest of the patient based on medical necessity OR (H) The patient has tried another drug in the same class and discontinued due to lack of efficacy/adverse event OR (I) There is support for use of the brand agent over generic (e.g., two null mutations in trans) AND
  • The prescriber is a specialist in metabolic disorders or has consulted with one AND
  • The patient will NOT use the agent in combination with another targeted agent included in this program AND
  • The patient does NOT have any FDA labeled contraindications to the requested agent AND
  • The requested dose is within FDA labeling for the indication

Reauthorization criteria

  • The patient has been previously approved through the plan’s PA process AND
  • The patient has had improvement or stabilization as follows:
  • For Kuvan or sapropterin: ONE of (A) Phe levels maintained within acceptable range [<12 years and females pregnant or planning: 120–360 micromol/L (2–6 mg/dL); ≥12 years: 120–600 micromol/L (2–10 mg/dL)] OR (B) ≥30% decrease in Phe level from baseline
  • For Palynziq: ONE of (A) Phe level ≤600 micromol/L (10 mg/dL) OR (B) ≥20% decrease in Phe level from baseline OR (C) Has not received 16 weeks at maximum dose and prescriber will evaluate for dose escalation AND
  • ONE of the following: (A) The patient is on a Phe-restricted diet and will continue OR (B) If Palynziq, phenylalanine level <360 micromol/L (6 mg/dL) AND
  • If the requested is brand, ONE of the following: (A) Stable on requested agent OR (B) Tried and had inadequate response to generic sapropterin OR (C) Has intolerance/hypersensitivity to generic not expected with brand OR (D) Has contraindication to generic not expected with brand OR (E) Generic discontinued due to inadequate effect or adverse event OR (F) Generic sapropterin expected ineffective or harmful or barrier to adherence OR (G) Generic not in best interest medically OR (H) Tried another drug in same class stopped for efficacy/adverse event OR (I) Support for brand over generic (e.g., two null mutations)
  • The prescriber is a specialist in metabolic disorders or consulted with one AND
  • No combination with another targeted agent AND
  • No FDA labeled contraindications AND
  • Dose within FDA labeling

Approval duration

12 months (BCBSIL); 3–9 months (BCBSMT/BCBSNM); 1–2 months (other plans depending on dose)