PDUFA date for AMONDYS 45 and VYONDYS 53 sNDAs (conversion to full approval) in DMD
The FDA is due to decide by Feb 28, 2027 whether to approve Sarepta’s AMONDYS 45 (casimersen) / VYONDYS 53 (golodirsen) for Duchenne muscular dystrophy (exon 45 / exon 53 skipping). Approval clears it for sale in the US; a rejection usually arrives as a letter listing what has to be fixed first.
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary
- Company
- Sarepta Therapeutics, Inc. SRPT
- Drug
- AMONDYS 45 (casimersen) / VYONDYS 53 (golodirsen)
- For
- Duchenne muscular dystrophy (exon 45 / exon 53 skipping)
- Stage
- sNDA under FDA review
- When
- Feb 28, 2027
- Source note
- FDA accepted sNDAs (announced July 1, 2026) seeking conversion of accelerated approvals to traditional approval, supported by ESSENCE confirmatory data and real-world evidence; PDUFA target action date Feb 28, 2027.
- Checked
- Checked by a person when it was added
- Source: investorrelations.sarepta.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- Drugs@FDA ↗ — search the drug name for approval letters
- SRPT headlines on this site
Sarepta expects to report results from its Phase 1/2 study of siRNA candidates (DM1 and FSHD programs) for Myotonic dystrophy type 1; facioscapulohumeral muscular dystrophy in the second half of 2026 (no exact date yet). Phase 1/2 is an early study that checks safety and takes a first look at effect.
More details
- Entry
- Phase 1/2 MAD-cohort readouts for siRNA programs in DM1 and FSHD · Clinical trial results (readout / topline data)
- Drug
- siRNA candidates (DM1 and FSHD programs)
- For
- Myotonic dystrophy type 1; facioscapulohumeral muscular dystrophy
- Stage
- Phase 1/2 · an early study that checks safety and takes a first look at effect
- When
- H2 2026 · the company gave a window, not a day
- Source note
- Per Q2 2026 earnings release (Aug 5, 2026): readouts from the multiple-ascending-dose cohorts of ongoing Phase 1/2 studies in DM1 and FSHD remain on track for 2H 2026.
- Checked
- Checked by a person when it was added
- Verify it
- Source: businesswire.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- SRPT headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page
ELEVIDYS ENDEAVOR Cohort 8 full enrollment expected by year-end 2026, expected in Q4 2026 (Oct–Dec) (no exact date yet). A company milestone for Sarepta (ELEVIDYS (delandistrogene moxeparvovec) for Duchenne muscular dystrophy): a step in the program rather than a decision or a data readout.
More details
- Entry
- ELEVIDYS ENDEAVOR Cohort 8 full enrollment expected by year-end 2026 · Company milestone
- Drug
- ELEVIDYS (delandistrogene moxeparvovec)
- For
- Duchenne muscular dystrophy
- Stage
- Phase 1b (ENDEAVOR Cohort 8)
- When
- Q4 2026 · the company gave a window, not a day
- Source note
- Q2 2026 earnings PR (Aug 5, 2026) guides to completing enrollment of ENDEAVOR Cohort 8 by year-end 2026, ahead of the already-tracked Q1 2027 12-week data readout.
- Checked
- Checked by a person when it was added
- Verify it
- Source: biospace.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- SRPT headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page
Sarepta expects to report results from its Phase 1b (ENDEAVOR Cohort 8) study of ELEVIDYS (delandistrogene moxeparvovec) + immunosuppressant regimen for Duchenne muscular dystrophy (gene therapy safety/immunosuppression cohort) in Q1 2027 (Jan–Mar) (no exact date yet). This is the first look at whether the drug did what the company hoped.
More details
- Entry
- ENDEAVOR Cohort 8 12-week data for ELEVIDYS with immunosuppression · Clinical trial results (readout / topline data)
- Drug
- ELEVIDYS (delandistrogene moxeparvovec) + immunosuppressant regimen
- For
- Duchenne muscular dystrophy (gene therapy safety/immunosuppression cohort)
- Stage
- Phase 1b (ENDEAVOR Cohort 8)
- When
- Q1 2027 · the company gave a window, not a day
- Source note
- Q2 2026 earnings release guides full enrollment of ENDEAVOR Cohort 8 by year-end 2026 and 12-week data from the full cohort in Q1 2027 — key to ELEVIDYS's path back to broader use after 2026 safety events and label restriction to ambulatory patients.
- Checked
- Checked by a person when it was added
- Verify it
- Source: businesswire.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- SRPT headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page