EXONDYS 51
EXONDYS 51 (eteplirsen) is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD). It is specifically for patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. The drug received accelerated approval based on observed increases in dystrophin in skeletal muscle, though continued approval may be contingent upon verification of clinical benefit in confirmatory trials.
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.
Details
- Status
- Prescription
- First Approved
- 2016-09-19
- 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
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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
ProIndications & Usage
FDA Label (PDF)EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51 [see Clinical Studies ] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. EXONDYS 51 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients...
EXONDYS 51 Patents & Exclusivity
Patents (28 active)
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.