News

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 ...
US FDA Investigates Third Death After Treatment With Sarepta's Gene Therapy Elevidys
(Reuters) -The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who ...
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 ...
FDA says it is investigating death of child who received Sarepta’s Elevidys
The U.S. Food and Drug Administration said on Friday that it is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics (NASDAQ:SRPT) gene therapy for Duchenne ...
Sarepta falls as FDA plans to investigate death of child who received Elevidys
The U.S. Food and Drug Administration said on Friday that it is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics ( NASDAQ: SRPT) gene therapy for Duchenne ...
Pharmaceutical Technology4h
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 Head To ‘Listen To Concerns’ Regarding Coerced Use Of Abortion Pill Widely Available Online
Commissioner Marty Makary said in an interview with Politico’s Dasha Burns that the FDA will “look at the data” surrounding ...
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.
Medscape9h
EMA Says No to Duchenne Gene TherapyElevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
Precision Medicine Online9h
EMA's CHMP Recommends Against Approval for Sarepta, Roche Duchenne Gene Therapy Elevidys
Elevidys, which is also facing regulatory hurdles in the US, was developed by Sarepta Therapeutics but is sold by Roche outside of the US.
Applied Clinical Trials Online9h
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.