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FDA Medication Tracker

← All approvals · Rare & genetic · 2025

Sephience (sepiapterin)

The FDA approved Sephience (sepiapterin) on July 28, 2025 to treat hyperphenylalaninemia in patients with sepiapterin-responsive phenylketonuria, in conjunction with a phenylalanine-restricted diet.
Brand name
Sephience
Generic name
sepiapterin
FDA approval date
July 28, 2025
Approved use (FDA wording)
To treat hyperphenylalaninemia in patients with sepiapterin-responsive phenylketonuria, in conjunction with a phenylalanine-restricted diet
Company
Ptc Therap
Application
NDA219666
Dosage form / route
POWDER · ORAL
Boxed warning on label
Not listed
Category on this site
Rare & genetic

Indications and usage, from the FDA label

SEPHIENCE is 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. 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. ( 1 )

Official sources

Facts only. This site lists what the FDA has approved and what the FDA says each drug is approved for, quoted or closely paraphrased from FDA and openFDA records. It gives no medical advice, no dosing, and no comparisons, and it says nothing about whether a drug is right for anyone. Talk to a licensed clinician about any medication. Records can lag or contain errors; the FDA's own pages are authoritative.