Sarepta Therapeutics, Inc. SRPT
Sarepta Therapeutics, Inc. (Nasdaq: SRPT) is a rare genetic drug developer. Its lead program, SRP-9003 (bidridistrogene xeboparvovec), is in Phase 3 for LGMD2E (beta-sarcoglycanopathy). Its next catalyst on record is VYONDYS 53 and AMONDYS 45: PDUFA target action date for sNDAs (February 2027).
Upcoming catalysts
- February 2027VYONDYS 53 and AMONDYS 45 — PDUFA target action date for sNDAs
As of the date of this Quarterly Report, we anticipate a PDUFA target action date of February 28, 2027.
Next further out: SRP-1003 (DM1) — Multiple ascending dose data (Q4 2026). 4 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| EXONDYS 51 (eteplirsen) | Duchenne muscular dystrophy (exon 51 amenable) | oligonucleotide-other | Approved |
| VYONDYS 53 (golodirsen) | Duchenne muscular dystrophy (exon 53 amenable) | oligonucleotide-other | Approved |
| AMONDYS 45 (casimersen) | Duchenne muscular dystrophy (exon 45 amenable) | oligonucleotide-other | Approved |
| ELEVIDYS (delandistrogene moxeparvovec-rokl) | Duchenne muscular dystrophy | gene therapy | Approved |
| SRP-9003 | LGMD2E (beta-sarcoglycanopathy) | gene therapy | Phase 3 |
| SRP-1003 | Myotonic dystrophy type 1 (DM1) | RNA (siRNA) | Phase 1 |
| SRP-1001 | Facioscapulohumeral muscular dystrophy (FSHD) | RNA (siRNA) | Phase 1 |
| SRP-1005 | Huntington's disease (HD) | RNA (siRNA) | Phase 1 |
Recent results and decisions
- May 6, 2026submitted Sarepta completed sNDA submissions to FDA seeking conversion of AMONDYS 45 and VYONDYS 53 from accelerated to traditional approval.
Completed submission of sNDA for AMONDYS 45 and VYONDYS 53 to FDA seeking conversion to traditional approval
Filing - May 6, 2026positive First Phase 1/2 data for SRP-1001 showed dose-dependent drug exposure, early biomarker effects and favorable tolerability.
siRNA pipeline delivers first Phase 1/2 data for SRP-1001 (FSHD1) and SRP-1003 (DM1) showing dose-dependent drug exposure, early biomarker effects, and favorable tolerability
Filing - May 6, 2026positive First Phase 1/2 data for SRP-1003 showed dose-dependent drug exposure, early biomarker effects and favorable tolerability.
siRNA pipeline delivers first Phase 1/2 data for SRP-1001 (FSHD1) and SRP-1003 (DM1) showing dose-dependent drug exposure, early biomarker effects, and favorable tolerability
Filing
3 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.