Ultragenyx Pharmaceutical
As of the edition of 20 October 2026, Ultragenyx Pharmaceutical runs 11 drugs, 5 of them marketed; it is on route A at 0 targets and on route B at 1 target.
Sources: ClinicalTrials.gov, FDA Orange Book and Purple Book, other approval registers, company records.
Family history
The records place 2 company names in this family.
Drugs by status
| Status | Count |
|---|---|
| Marketed | 5 |
| Active | 3 |
| Went quiet | 1 |
| Stopped | 2 |
| Total | 11 |
Marketed drugs and when their US protection ends
| Drug | US protection | Route B rows |
|---|---|---|
| Triheptanoin | Ends 2027 (exclusivity) | 0 |
| Evinacumab | Ends 2033 (computed from the first US licence) | 2 |
| Vestronidase alfa | Ends 2029 (computed from the first US licence) | 0 |
| Rebisufligene etisparvovec | Ends 2038 (computed from the first US licence) | 0 |
| Pariglasgene brecaparvovec | Ends 2038 (computed from the first US licence) | 0 |
A route B row is one company at one target.
Where it needs a new molecule
Route A at 0 targets, route B at 1 target.
| Target | Route |
|---|---|
| ANGPTL3 Angiopoietin-related protein 3 | Route B |
All 1 row on route A or B
In the product1 company-target row on route A or B; 0 are not shown here, and no score is.
See all 1 targets where Ultragenyx Pharmaceutical needs a new molecule, with the facts behind eachRequest access. Nothing is shown until you do.
Where its trials are
- Inherited and neonatal: 20 trials led
- Skin and connective tissue: 14 trials led
- Nutrition and metabolism: 10 trials led