News

FDA opens probe into mysterious death linked to gene therapy
The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...
What’s behind Sarepta’s gene therapy deaths?
Three patient deaths linked to gene therapies developed by Sarepta Therapeutics (SRPT) have drawn FDA scrutiny on the viral ...
Medscape11h
EMA Says No to Duchenne Gene Therapy Elevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
PipelineReview.com13h
Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU
BASEL, Switzerland I July 25, 2025 I Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the ...
EconoTimes21h
FDA Probes Death of 8-Year-Old Linked to Sarepta’s Elevidys Gene Therapy
The U.S. Food and Drug Administration (FDA) is investigating the death of an eight-year-old boy who received Sarepta ...
FDA Probes Death of Boy in Brazil on Sarepta’s Elevidys
US regulators are investigating the death of an 8-year-old boy in Brazil who received Sarepta Therapeutics Inc.’s Elevidys.
Sarepta Therapeutics Provides Clarifying Statement on ELEVIDYS
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the ...
FDA probes death of patient on Sarepta's Elevidys, partner Roche says death unrelated to therapy
The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received ...
Sarepta fails to win EU backing for muscle disorder gene therapy
Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...
Pharmaceutical Technology1d
Sarepta refuses to pull gene therapy despite FDA request
Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the FDA.
FDA chief talks staffing, abortion drug and tobacco
The FDA is still on track to meet its user fee targets despite a higher-than-normal vacancy rate among scientific reviewers.
Applied Clinical Trials Online1d
FDA Crackdown on Trial Design: What July’s CRLs to Replimune and Capricor Mean for Sponsors
Complete Response Letters recently issued by the FDA signal heightened scrutiny of trial design and reinforce the agency’s shifting regulatory expectations for sponsors and CROs.