Quick answers
- What is Sepiapterin used for?
- The FDA-approved label (Sephience, PTC Therapeutics Inc., April 24, 2026) says: “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. …” This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- Does Sepiapterin have a boxed warning?
- No. None of the 2 FDA labels we found for this medicine has a boxed warning.
- Is there a shortage of Sepiapterin?
- No current shortage is listed by the FDA for the products of this medicine (FDA data from October 30, 2026).
- Has Sepiapterin been recalled?
- No recall linked to the products of this medicine appears in the FDA enforcement reports we use.
- Who makes Sepiapterin?
- 2 companies list prescription products of Sepiapterin in the FDA NDC Directory, including PTC Therapeutics, Inc. and allphamed Pharbil Arzneimittel GmbH.
What Sepiapterin is used for
Quoted from the “Indications and Usage” section of the FDA-approved 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. …
— FDA label: Sephience, PTC Therapeutics Inc., effective April 24, 2026. Read the full label on DailyMed →
The FDA has 2 labels for products of this medicine (different makers and forms); uses can differ between them. Check the label that comes with your own medicine.
This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
Boxed warning
A boxed warning (“black box”) is the FDA’s strongest warning on a drug label, for serious or life-threatening risks.
The label quoted above has no boxed warning.
0 of 2 FDA labels for this medicine have a boxed warning.
Forms and how it is given
| Dosage forms | Powder |
|---|---|
| Route | Oral |
| Prescription products | 4 in the FDA NDC Directory |
| Brand names | Sephience |
Companies that list this medicine
Labelers in the FDA NDC Directory: the company named on the package (maker or distributor), with the number of prescription products.
- PTC Therapeutics, Inc.2 products
- allphamed Pharbil Arzneimittel GmbH2 products
FDA approval history
- NDA: New Drug Application: how a new (usually brand-name) medicine is approved.
| Application | Sponsor | Approved | Later changes approved |
|---|---|---|---|
| NDA219666 SEPHIENCE | Ptc Therap | July 28, 2025 | — |
Shortage status
The FDA drug shortage list does not show these products.
Recalls
No recall linked to these products (by their NDC code) appears in the FDA enforcement reports.
Official identifiers
| RxCUI (RxNorm) | 2723061, 2723067, 2723069, 2723071 |
|---|---|
| SPL set ID of the quoted label | 32ac719c-49f0-4105-9c46-18c19583a5c2 |
| FDA labels (SPL) | 2 |
| FDA applications | NDA219666 |
| Active ingredients (FDA) | SEPIAPTERIN |
Sources
- FDA-approved label on DailyMed (National Library of Medicine) — Sephience, PTC Therapeutics Inc., effective April 24, 2026; via openFDA, FDA data exported October 30, 2026, retrieved October 31, 2026
- NDC Directory (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drugs@FDA (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drug recall enforcement reports (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 27, 2026, retrieved October 31, 2026
- Drug shortages (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
openFDA data is provided by the FDA as is; it may be incomplete. Always rely on the label that comes with your medicine. FDA Enforcement Report search →