pegvaliase-pqpz — Blue Cross Blue Shield of New Mexico

Initial criteria

• 1. The patient has a diagnosis of phenylketonuria (PKU) AND
• 2. 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
• 3. ONE of the following: (A) BOTH of the following: (1) Phenylalanine levels cannot be maintained within the recommended maintenance range with dietary intervention (phenylalanine-restriction) 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
• 4. If the requested agent is Kuvan or sapropterin, then ONE of the following: (A) The patient is less than 12 years of age 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 currently pregnant AND has a baseline Phe level >360 micromol/L (6 mg/dL) AND
• 5. If the requested agent is Palynziq, the patient has a baseline blood Phe level >600 micromol/L (10 mg/dL) AND
• 6. If the request is for a brand agent, then ONE of the following: (A) The patient is currently being treated with the requested agent and stable OR (B) Tried and had inadequate response to generic sapropterin OR (C) Intolerance or hypersensitivity to generic sapropterin not expected with brand OR (D) FDA labeled contraindication to generic sapropterin not expected with brand OR (E) Generic sapropterin discontinued due to lack of efficacy/adverse event OR (F) Generic sapropterin expected to be ineffective due to patient characteristics or causes harm/adherence issues OR (G) Generic sapropterin not in the best interest of the patient based on medical necessity OR (H) Tried another drug in same class as generic sapropterin with lack of efficacy/adverse event OR (I) Support for brand over generic sapropterin (e.g., two null mutations in trans) AND
• 7. The prescriber is a metabolic specialist or has consulted with one AND
• 8. The patient will NOT be using the requested agent in combination with another targeted agent included in this program AND
• 9. The patient does NOT have any FDA labeled contraindications to the requested agent AND
• 10. The requested quantity (dose) is within FDA labeled dosing for the requested indication

Reauthorization criteria

• 1. The patient has been previously approved for the requested agent through the plan’s prior authorization process AND
• 2. The patient has improvements or stabilization as indicated by: (A) For Kuvan/sapropterin: (1) Blood Phe levels 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) For Palynziq: (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 recommended dose and prescriber will evaluate for dose escalation AND
• 3. ONE of the following: (A) The patient is on and will continue a Phe-restricted diet OR (B) If Palynziq, the patient’s phenylalanine level <360 micromol/L (6 mg/dL) AND
• 4. If brand agent requested, one of the brand exception criteria (A–I in initial criteria) is met AND
• 5. Prescriber is a metabolic specialist or has consulted with one AND
• 6. The patient will NOT be using another targeted agent included in this program AND
• 7. No FDA labeled contraindications to requested agent AND
• 8. Requested dose within FDA labeled range

Approval duration

Kuvan: 2mo (initial 5–<20 mg/kg/day) or 1mo (≥20 mg/kg/day); Palynziq: 9mo; Sephience: 2mo (7.5–<60 mg/kg/day) or 1mo (≥60 mg/kg/day); renewals 12mo; BCBSNM Kuvan: 3mo, Palynziq: 9mo, Sephience: 3mo