Product Description
Drisapersen, (previously GSK2402968/PRO051) an antisense oligonucleotide, which induces exon skipping of exon 51, is currently in late stage development for DMD. (Sourced from: https://www.gsk.com/en-gb/media/press-releases/glaxosmithkline-s-drisapersen-previously-gsk2402968pro051-to-receive-food-and-drug-administration-breakthrough-therapy-designation/)
Mechanisms of Action: Exon51 Inducer
Novel Mechanism: Yes
Modality: Nucleic Acid
Route of Administration: Subcutaneous,Intravenous
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: Saudi Arabia
Approved Indications: None
Known Adverse Events: None
Company: GlaxoSmithKline
Company Location: BRENTFORD MIDDLESEX X0 TW8 9GS
Company CEO: Emma Walmsley
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Highest Development Phases
Phase 3: Muscular Dystrophy, Duchenne
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| Drisapersen extension study | P3 |
Terminated |
Muscular Dystrophy, Duchenne |
2019-06-08 |
|
| Drisapersen confirmatory study | P3 |
Completed |
Muscular Dystrophy, Duchenne |
2016-09-09 |
|
| 2007-004819-54 | P2 |
Completed |
Muscular Dystrophy, Duchenne |
2016-09-09 |
|
| NCT01910649 | P2 |
Terminated |
Muscular Dystrophy, Duchenne |
2016-09-01 |