Nitisinone
As of the edition of 20 October 2026, Nitisinone is a small molecule: marketed, first approved in 2002 (US); acts on HPD; no US protection of its own is listed.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2022
- Kind
- Small molecule
- Brand name
- Orfadin
- First approved
- 18 January 2002 (US)
- Approvals on record
- 18 in 5 regions
- Holder of the most recent approval
- Cycle Pharmaceuticals; 5 holders in all
- Registered trials
- 19, 1 stopped early
- Disease areas
- Nutrition and metabolism; Inherited and neonatal; Nervous system; Eye; Blood and lymph; Bones, joints and muscles
US protection
No US protection of its own is listed.
The FDA registers list no drug-substance patent or exclusivity of its own that counts for this drug.
Cycle Pharmaceuticals holds a later exclusivity on its own new formulation or combination product, ending 2032; it does not protect the original drug.
This is the US only.
Companies that need a new molecule because of this drug
2 companies on route B because of this drug.
Companies on route B for this drug
In the product2 companies are on route B because of Nitisinone.
See the 2 companies on route B for this drug, with the facts behind eachRequest access. Nothing is shown until you do.
Target
| Gene | Protein | UniProt |
|---|---|---|
| HPD | 4-hydroxyphenylpyruvate dioxygenase | P32754 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase not stated | 5 | 3 | 0 | 1 |
| Phase 1 | 7 | 7 | 0 | 0 |
| Phase 2 | 4 | 4 | 0 | 0 |
| Phase 3 | 3 | 2 | 0 | 0 |
| All trials | 19 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Swedish Orphan Biovitrum | Applicant, Acquired holder and Lead sponsor of trials | 2005 to now |
| Astellas | Applicant | 2014 to now |
| Cycle Pharmaceuticals | Later holder and Lead sponsor of trials | 2015 to now |