FDA requested Sarepta Therapeutics to suspend Elevidys distribution and placed clinical trials on hold due to safety concerns including three deaths in limb girdle muscular dystrophy trials; revoked platform technology designation.1
Elevidys holds full approval for ambulatory DMD patients aged 4+ (June 2024), but FDA plans to restrict non-ambulatory use pending confirmatory data amid new safety issues.12
Sarepta's Phase 3 ESSENCE trial failed for exon-skipping drugs Amondys 45 and Vyondys 53, yet company pursues full FDA approval with a meeting scheduled for February/March 2026.3
No recent news found on Collegium's $650M ADHD acquisition in provided results.
DMD pipeline active:
Advocates predict 2026 approvals for therapies like Dyne's DYNE-251 (exon 51 skipping, accelerated approval submission in 2026), Avidity's delpacibart zotadirsen, and Capricor's deramiocel (Phase 3 success, resubmission expected).245
Sources:
1. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold
2. https://musculardystrophynews.com/news/advocates-expect-exciting-year-ahead-duchenne-md-community/
3. https://www.biospace.com/business/after-mortality-marred-year-sarepta-looks-ahead-to-catalyst-rich-2026
4. https://cureduchenne.org/research/dyne-plans-to-submit-for-fda-accelerated-approval-for-duchenne-exon-51-skipping-in-2026-trial-still-recruiting-participants/
5. https://www.biospace.com/drug-development/following-sarepta-woes-others-look-to-unlock-the-next-chapter-in-dmd-treatment