Eteplirsen
As of the edition of 20 October 2026, Eteplirsen is an oligonucleotide: marketed, first approved in 2016 (US); acts on DMD; no US protection is listed.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2016
- Kind
- Oligonucleotide
- Brand name
- Exondys 51
- First approved
- 19 September 2016 (US)
- Approvals on record
- 2 in 1 region
- Holder of the most recent approval
- Avi Biopharma
- Registered trials
- 12, 1 stopped early
- Disease areas
- Bones, joints and muscles; Inherited and neonatal; Nervous system
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.
Target
| Gene | Protein | UniProt |
|---|---|---|
| DMD | Dystrophin | P11532 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase not stated | 1 | 0 | 1 | 0 |
| Phase 2 | 9 | 8 | 0 | 1 |
| Phase 3 | 2 | 1 | 1 | 0 |
| All trials | 12 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Avi Biopharma | Originator and Lead sponsor of trials | 2009 to now |