EXONDYS 51 (eteplirsen)
EXONDYS 51 is indicated for the treatment of Duchenne Muscular Dystrophy.
How EXONDYS 51 Works
Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in the exclusion of this exon during mRNA processing. This mechanism, known as exon skipping, is intended for patients with specific genetic mutations amenable to this process. By skipping exon 51, the drug allows for the production of an internally truncated dystrophin protein.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2016-09-19
- Patent Cliff
- 2034
- Routes
- INTRAVENOUS
- Dosage Forms
- SOLUTION
EXONDYS 51 Approval History
What EXONDYS 51 Treats
1 indicationsEXONDYS 51 is approved for 1 conditions since its original approval in 2016. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Duchenne Muscular Dystrophy
EXONDYS 51 Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in EXONDYS 51's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications EXONDYS 51 treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to EXONDYS 51
3 of 9FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
7 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT03992430 MIS51ON | 4658-402 2018-001762-42, 2024-511492-15-00 | Ph 3 | active not recruiting | A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) |
| NCT04179409 results posted | SRPT-Dup-US-001 | Ph 2 | completed | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. |
| NCT03985878 results posted | 4658-102-OLE 2019-000337-39 | Ph 2 | terminated | A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995) |
| NCT03218995 results posted | 4658-102 | Ph 2 | completed | Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping |
| NCT02420379 results posted | 4658-203 | Ph 2 | completed | Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy |
| NCT02255552 PROMOVI results posted | 4658-301 | Ph 3 | completed | Study of Eteplirsen in DMD Patients |
| NCT02286947 results posted | 4658-204 | Ph 2 | completed | Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
EXONDYS 51 FDA Label Details
Indications & Usage
FDA Label (PDF)EXONDYS 51 is indicated for the treatment of Duchenne Muscular Dystrophy.
EXONDYS 51 Patents & Exclusivity
Patents (5 active)
Pro Intelligence Preview
Deep insights for EXONDYS 51
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2034
- • 28 active patents
Trial Analysis
- • 7 total trials
- • Stage: Declining
Competitive Landscape
- • 9 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment