FDA accelerated approval of Exondys 51
The FDA approved Sarepta’s Exondys 51 (eteplirsen) for DMD on Sep 19, 2016. The company can now sell it in that market.
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary
- Company
- Sarepta Therapeutics, Inc. SRPT
- Drug
- Exondys 51 (eteplirsen)
- For
- DMD
- When
- Sep 19, 2016
- Source note
- First approved exon-skipping therapy for Duchenne muscular dystrophy.
- Checked
- Checked by a person when it was added
- 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
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.
More details
- Entry
- PDUFA date for AMONDYS 45 and VYONDYS 53 sNDAs (conversion to full approval) in DMD · FDA decision due (PDUFA date)
- 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
- Verify it
- 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
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page