Sepofarsen
As of the edition of 20 October 2026, Sepofarsen is an oligonucleotide: stopped in 2022; acts on CEP290.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Stopped, 2022
- Kind
- Oligonucleotide
- Registered trials
- 4, 1 stopped early
- Disease areas
- Eye; Inherited and neonatal; Nervous system; Nutrition and metabolism
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
1 company on route A because of this drug.
Companies on route A for this drug
In the product1 company is on route A because of Sepofarsen.
See the 1 company on route A for this drug, with the facts behind eachRequest access. Nothing is shown until you do.
Target
| Gene | Protein | UniProt |
|---|---|---|
| CEP290 | Centrosomal protein of 290 kDa | O15078 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase 2 | 2 | 1 | 0 | 1 |
| Phase 3 | 2 | 0 | 1 | 0 |
| All trials | 4 |
Who ran its trials
| Company | Role | Years |
|---|---|---|
| Laboratoires Thea | Lead sponsor of trials | 2017 to now |
| ProQR Therapeutics | Lead sponsor of trials | 2019 to now |