Product Description
a drug that acts to promote dystrophin production by restoring the translational reading frame of DMD through specific skipping of exon 51 in defective gene variants. Eteplirsen is applicable for approximately 14% of patients with DMDmutations. (Sourced from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5338848/)
Mechanisms of Action: mRNA Inhibitor
Novel Mechanism: No
Modality: Nucleic Acid
Route of Administration: Intravenous
FDA Designation: None *
Approval Status: Approved
Approved Countries: European Medicines Agency | Greece | Israel | Slovenia | United Kingdom | United States
Approved Indications: None
Known Adverse Events: None
Company: Sarepta
Company Location: CAMBRIDGE MA 02142
Company CEO: Douglas S. Ingram
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Colombia, Czech Republic, Denmark, France, Germany, Greece, Hungary, India, Ireland, Italy, Jordan, Korea, Mexico, Netherlands, New Zealand, Norway, Poland, Romania, Serbia, Slovenia, Spain, Switzerland, Taiwan, Turkey, United Kingdom, United States
Active Clinical Trial Count: 4
Highest Development Phases
Phase 3: Muscular Dystrophy, Duchenne
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| 4658-402 | P3 |
Unknown Status |
Muscular Dystrophy, Duchenne |
2026-10-31 |
|
| MIS51ON | P3 |
Active, not recruiting |
Muscular Dystrophy, Duchenne |
2026-10-31 |
|
| 2018-001762-42 | P3 |
Active, not recruiting |
Muscular Dystrophy, Duchenne |
2026-01-03 |
|
| 2016-005024-28 | P2 |
Active, not recruiting |
Muscular Dystrophy, Duchenne |
2019-06-18 |