Drisapersen
As of the edition of 20 October 2026, Drisapersen is an oligonucleotide: stopped in 2016; acts on DMD.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Stopped, 2016
- Kind
- Oligonucleotide
- Registered trials
- 9, 4 stopped early
- Disease areas
- Bones, joints and muscles; Inherited and neonatal; Nervous system
US protection
No US approval on record.
With no US approval on record there are no listed US patents or exclusivities.
This is the US only.
Companies that need a new molecule because of this drug
2 companies on route A because of this drug.
Companies on route A for this drug
In the product2 companies are on route A because of Drisapersen.
See the 2 companies on route A for this drug, with the facts behind eachRequest access. Nothing is shown until you do.
Target
| Gene | Protein | UniProt |
|---|---|---|
| DMD | Dystrophin | P11532 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase not stated | 1 | 0 | 0 | 0 |
| Phase 1 | 1 | 1 | 0 | 0 |
| Phase 2 | 3 | 2 | 0 | 1 |
| Phase 3 | 4 | 1 | 0 | 3 |
| All trials | 9 |
Who ran its trials
| Company | Role | Years |
|---|---|---|
| GSK | Lead sponsor of trials | 2010 to now |
| Amicus Therapeutics | Lead sponsor of trials |