PAH
Phenylalanine-4-hydroxylase
As of the edition of 20 October 2026, PAH (phenylalanine-4-hydroxylase, UniProt P00439) has 2 drugs tested at it and 2 companies on the list, 0 on route A and 2 on route B.
Sources: UniProt, ClinicalTrials.gov, ChEMBL, FDA Orange Book and Purple Book, Pharos.
The protein
- UniProt
- P00439
- How well studied
- Drug target: an approved drug acts on it
- Solved structures
- 21
- Disease areas
- Inherited and neonatal; Nervous system; Nutrition and metabolism; Heart and blood vessels; Mental health; Lungs and airways; Digestive system; Pregnancy and women's health
- Registered trials of its drugs
- 77
Drugs at this target
| Status | Count |
|---|---|
| Marketed | 2 |
| Total | 2 |
| Drug | Status | Furthest phase | US protection |
|---|---|---|---|
| Sapropterin | Marketed | Phase 4 | none listed |
| Sepiapterin | Marketed | Phase 3 | Ends 2038 |
2 of 2 drugs, the most tested first.
Companies at this target
| Where the company stands | Companies |
|---|---|
| Route B | 2 |
| Total on the list | 2 |
| Company | Route |
|---|---|
| Amicus Therapeutics | Route B |
| PTC Therapeutics | Route B |
The other 0 companies at PAH
In the product2 companies in all; 0 are not named here, and no score is.
See the 2 companies at PAH, ranked, with the facts behind eachRequest access. Nothing is shown until you do.