EXONDYS 51 (eteplirsen)
Duchenne Muscular Dystrophy (DMD)
MarketedFDA Approved
Key Facts
Indication
Duchenne Muscular Dystrophy (DMD)
Phase
Marketed
Status
FDA Approved
Company
About sarepta-therapeutics
Sarepta Therapeutics is a commercial-stage biotech leader with an urgent mission to engineer and deliver precision genetic medicines for rare diseases. The company has achieved significant commercial success with multiple FDA-approved RNA-based exon-skipping therapies for Duchenne muscular dystrophy (DMD) and the landmark approval of ELEVIDYS, a gene therapy for DMD. Its strategy leverages a deep, multi-modal pipeline, a hybrid manufacturing model for scale, and a global commercial footprint to solidify its position as a dominant force in rare disease therapeutics.
View full company profileTherapeutic Areas
Other Duchenne Muscular Dystrophy (DMD) Drugs
| Drug | Company | Phase |
|---|---|---|
| DT-DEC01 | Dystrogen Therapeutics | Phase 1 |
| Skeletal Muscle Regeneration Program | IPS HEART | Pre-clinical |
| KYMBEE (deflazacort) | Upsher-Smith | Approved |
| ALY688ER | Allysta Pharmaceuticals | Phase 1 |
| AVGN7 + AVGND | AAVogen | Pre-clinical |
| Myomatrix Stabilization Program | strykagen | Preclinical |
| RIPPLE™ (implied) | sonothera | Preclinical |
| Vamorolone (AGAMREE) | reveragen-biopharma | Approved |
| AGAMREE (vamorolone) | catalyst-pharmaceuticals | Marketed |
| EDG-5506 | edgewise-therapeutics | Phase 2 |
| ELEVIDYS (delandistrogene moxeparvovec-rokl) | sarepta-therapeutics | Marketed / Phase 3 |
| SRP-5051 (vesleteplirsen) | sarepta-therapeutics | Phase 2 |