Sapropterin
As of the edition of 20 October 2026, Sapropterin is a small molecule: marketed, first approved in 2007 (US); acts on PAH; no US protection is listed.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2010
- Kind
- Small molecule
- Brand name
- Kuvan
- First approved
- 13 December 2007 (US)
- Approvals on record
- 7 in 4 regions
- Holder of the most recent approval
- Amicus Therapeutics; 3 holders in all
- Registered trials
- 72, 13 stopped early
- Disease areas
- Inherited and neonatal; Nervous system; Nutrition and metabolism; Heart and blood vessels; Mental health; Lungs and airways
US protection
No US protection is listed.
The FDA registers list no drug-substance patent or exclusivity that counts for this drug.
This is the US only.
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 Sapropterin.
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 | 19 | 12 | 2 | 4 |
| Phase 1 | 13 | 7 | 2 | 2 |
| Phase 2 | 23 | 16 | 1 | 4 |
| Phase 3 | 12 | 10 | 0 | 2 |
| Phase 4 | 5 | 4 | 0 | 1 |
| All trials | 72 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Amicus Therapeutics | Originator, Applicant and Lead sponsor of trials | 2007 to now |