AMONDYS 45 (casimersen) · Sarepta Therapeutics
AMONDYS 45 (casimersen) is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD). It is specifically used for patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. The drug received accelerated approval based on its ability to increase dystrophin production in skeletal muscle, though continued approval may be contingent upon verification of clinical benefit in confirmatory trials.
How AMONDYS 45 Works
Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA, which results in the exclusion of this exon during mRNA processing. This exon skipping is intended to allow for the production of an internally truncated dystrophin protein in patients with specific genetic mutations. By facilitating the creation of this truncated protein, the drug addresses the underlying genetic deficiency associated with the disease.
Details
- Status
- Prescription
- First Approved
- 2021-02-25
- Patent Cliff
- 2030
- Routes
- INTRAVENOUS
- Dosage Forms
- SOLUTION
AMONDYS 45 Approval History
What AMONDYS 45 Treats
1 indicationsAMONDYS 45 is approved for 1 conditions since its original approval in 2021. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Duchenne Muscular Dystrophy
AMONDYS 45 Competitive Set
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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 AMONDYS 45's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications AMONDYS 45 treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to AMONDYS 45
3 of 9FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Sarepta Therapeutics's other novel FDA approvals
Other CDER-designated drugs from the same sponsor (2016–2025).
Clinical Trial Registry
2 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT03532542 results posted | 4045-302 2017-004625-32 | Ph 3 | terminated | An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy |
| 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. |
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.
AMONDYS 45 FDA Label Details
Indications & Usage
FDA Label (PDF)AMONDYS 45 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 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45 [see Clinical Studies ] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. AMONDYS 45 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in pa...
AMONDYS 45 Patents & Exclusivity
Patents (5 active)
Exclusivity
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Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2030
- • 7 active patents
Trial Analysis
- • Clinical trial tracking
- • Development stage analysis
Competitive Landscape
- • 9 similar drugs
- • Same target/indication analysis
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.