Sepiapterin
As of the edition of 20 October 2026, Sepiapterin is a small molecule: marketed, first approved in 2025 (EU); acts on PAH; US protection ends in 2038.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2026
- Kind
- Small molecule
- Brand name
- Sephience
- First approved
- 19 June 2025 (EU)
- Approvals on record
- 6 in 4 regions
- Holder of the most recent approval
- PTC FARMACEUTICA DO BRASIL LTDA.; 4 holders in all
- Registered trials
- 6, 0 stopped early
- Disease areas
- Inherited and neonatal; Nervous system; Nutrition and metabolism; Digestive system
US protection
US protection ends in 2038.
The last listed US drug-substance patent ends in 2038. The application that gives this date is held by PTC Therapeutics.
This is the US only, and it is not the date generics or biosimilars can enter.
The exact date and the listings behind it
In the product6 listings count towards this date. The exact date and each listing are in the product.
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Companies that need a new molecule because of this drug
1 company on route B because of this drug.
Companies on route B for this drug
In the product1 company is on route B because of Sepiapterin.
See the 1 company on route B for this drug, with the facts behind eachRequest access. Nothing is shown until you do.
Target
| Gene | Protein | UniProt |
|---|---|---|
| PAH | Phenylalanine-4-hydroxylase | P00439 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase not stated | 1 | 0 | 1 | 0 |
| Phase 2 | 2 | 2 | 0 | 0 |
| Phase 3 | 3 | 2 | 1 | 0 |
| All trials | 6 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| PTC Therapeutics | Applicant and Lead sponsor of trials | 2019 to now |