Sephience™

Drug - Sephience™ (sepiapterin) [PTC Therapeutics, Inc.]

May 2026

Therapeutic Area - Phenylketonuria

Initial approval criteria

  • Age ≥ 1 month; AND
  • Diagnosis of phenylketonuria (PKU); AND
  • Phenylalanine (Phe) levels cannot be maintained within the recommended maintenance range with dietary intervention (Phe restriction); AND
  • Phe-restricted diet will continue during treatment with Sephience; AND
  • Prescriber has obtained blood Phe levels at baseline and will continue to monitor as clinically appropriate; AND
  • Patient has tried and had an inadequate response to generic sapropterin despite monitored adherence to treatment; OR
  • Patient has an intolerance or hypersensitivity to generic sapropterin that is not expected to occur with Sephience; OR
  • Patient has an FDA labeled contraindication to generic sapropterin that is not expected to occur with Sephience; AND
  • Patient will NOT use Sephience in combination with another targeted agent for PKU (e.g., sapropterin [Kuvan, Javygtor], pegvaliase-pqpz [Palynziq]); AND
  • The prescriber is a specialist in the area of the patient’s diagnosis (e.g., metabolic disorders) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis.
  • Initial approval:
    • 2 months if initial dose is 7.5 mg/kg/day to < 60 mg/kg/day
    • 1 month if initial dose is 60 mg/kg/day

Renewal criteria

  • Patient must continue to meet the above criteria; AND
  • Patient must have disease improvement and/or stabilization (e.g., blood Phe levels within an acceptable range, decrease in blood Phe levels from baseline); AND
  • Patient has not experienced any treatment-restricting adverse effects (e.g., active bleeding).
  • Renewal approval is for 12 months

Quantity limits

  • Max dose 60 mg/kg/day
  • Patient’s weight (in kg) must be submitted at time of request
  • When a combination of the two Sephience strengths must be prescribed, the prescriber must provide:
    • Attestation that two separate prescriptions for two Sephience strengths will be issued AND
    • The requested quantity, number of refill(s), and directions for use for each Sephience strength on the prior authorization request form AND
    • Attestation that when patient becomes stabilized on one Sephience strength, reasonable efforts will be taken to ensure that the other Sephience strength will be used up to minimize waste

Background

Sephience is a phenylalanine hydroxylase (PAH) activator indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). Sephience is to be used in conjunction with a phenylalanine (Phe)- restricted diet.

Questions

Provider Call Center: (844) 575-7887