News

FDA probes deaths of boys taking Sarepta Duchenne drug
The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...
No Accelerated FDA Approval For Edgewise's Rare Muscular Disease Drug; Shares Fall
Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...
Edgewise falls after data for muscular dystrophy therapy
Edgewise Therapeutics (NASDAQ:EWTX) lost ~9% in the morning hours on Thursday after the company posted data from clinical ...
Fierce Pharma1d
FDA investigates patient deaths after treatment with Sarepta's Duchenne gene therapy Elevidys
In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
FDA has indicated that an Advisory Committee meeting is not required at this timeIn-person late-cycle review meeting ...
BioSpace1d
FDA Reviews Sarepta’s Elevidys After Second Patient Death
The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...
MedPage Today on MSN1d
FDA Probes Gene Therapy After Two Deaths
The FDA is investigating the deaths of two boys with Duchenne muscular dystrophy who died soon after being treated with ...
STAT8dOpinion
Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys
Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...
STAT1d
Pharmalittle: We’re reading about FDA probing deaths after Sarepta treatment, a Regeneron plan for charities, and more
The FDA is investigating two deaths in non-ambulatory Duchenne muscular dystrophy patients who received a Sarepta gene ...
BioSpace17h
Novavax Approval Process a Potential ‘Harbinger’ of Threat to FDA Integrity: Former Regulators
Writing in JAMA, four former governmental officials warn that the Trump administration’s involvement in delaying the approval ...
FDA Skips Advisory Committee For Capricor's Duchenne Cell Therapy
Capricor's DMD therapy Deramiocel is under FDA Priority Review with no major issues flagged; PDUFA date set for August 31, 2025.
FDA investigates patient deaths after treatment with Sarepta's gene therapy
The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...