May 14, 2026
A U.S. appeals court revived Regenxbio’s patent suit against Sarepta, holding the asserted gene-therapy host cell claims are markedly different from anything occurring in nature.
Apr 23, 2026
Sarepta Therapeutics said it will announce first quarter 2026 financial results. The event will be webcast on its investor relations website, with a replay archived for one year.
Mar 23, 2026
Sarepta Therapeutics received FDA feedback supporting supplemental applications for AMONDYS 45 and VYONDYS 53, while New Zealand approved a Phase 1 trial for Huntington's Disease candidate SRP-1005. The company faces significant share price declines despite these developments, with analysts showing wide disagreement on valuation targets ranging from $5 to $80.
Feb 28, 2026
Chugai Pharmaceutical launched ELEVIDYS in Japan as the first regenerative medical product for Duchenne muscular dystrophy following its listing on the National Health Insurance reimbursement price list on February 20, 2026.
Feb 12, 2026
PTC Therapeutics has withdrawn its New Drug Application resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy following FDA feedback that the data are unlikely to meet the threshold for substantial evidence of effectiveness.
Feb 17, 2026
Ultragenyx Pharmaceutical confronts multiple shareholder class action lawsuits over Phase III trial disclosures while reporting positive UX111 gene therapy data and implementing a 10% workforce reduction.
Feb 13, 2026
PTC Therapeutics withdrew its resubmitted new drug application for Translarna in Duchenne muscular dystrophy after the FDA indicated the data was unlikely to meet the threshold for substantial evidence of effectiveness, marking the third unsuccessful US approval attempt.