Dystrophin pre-mRNA Inhibitors
4 drugsAbout Dystrophin pre-mRNA
Dystrophin pre-mRNA is the precursor to mature mRNA encoding dystrophin, a protein vital for muscle fiber integrity. Mutations disrupt pre-mRNA splicing, leading to non-functional dystrophin and Duchenne muscular dystrophy (DMD). Targeting pre-mRNA aims to restore the reading frame and produce partially functional dystrophin.
Dystrophin pre-mRNA is a therapeutic target for DMD, a rare disease. There is currently no genetic evidence data available linking variations in the pre-mRNA sequence to disease severity.
Four FDA-approved drugs (EXONDYS 51, VYONDYS 53, AMONDYS 45, VILTEPSO) target dystrophin pre-mRNA using antisense oligonucleotides (ASO). These drugs are developed by Sarepta Therapeutics and Nippon Shinyaku for rare disease indications.
Top Dystrophin pre-mRNA Drugs
Sarepta Therapeutics and Nippon Shinyaku are the only companies with approved drugs.
High market concentration indicates potential entry barriers or partnership opportunities.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| AMONDYS 45 | Sarepta Therapeutics | 2021 | 1 |
Dystrophin pre-mRNA Drug Modality Landscape
Modalities
Routes of Administration
Dystrophin pre-mRNA requires biologic approaches (aso), likely due to its structure or location.
Explore alternative modalities to differentiate from existing ASO therapies.
Dystrophin pre-mRNA Clinical Trials 14 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on Dystrophin pre-mRNA. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 2 | 8 | 7 | 1 | 0 | 88% |
| Phase 3 | 4 | 2 | 1 | 1 | 67% |
| Phase 4 | 2 | 0 | 1 | 1 | 0% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Dystrophin pre-mRNA Drug Approval Timeline (2016 - 2021)
The first drug was approved in 2016, and the most recent in 2021.
Recent approvals suggest continued interest, but approaching market saturation.
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Competitive Landscape
- • 2 companies competing
- • Market share by company
Full Drug Portfolio
- • All 4 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 4-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 4 (2 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on Dystrophin pre-mRNA. A trial counts if an approved drug acting on Dystrophin pre-mRNA is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.