News

Fierce Pharma2d
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, ...
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 ...
BioSpace3d
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 ...
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 ...
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 ...
BioSpace1d
Edgewise Takes a Hit as FDA Blocks Accelerated Path for Becker Muscular Dystrophy Drug
The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for ...
Stocktwits on MSN3d
Capricor Therapeutics Says FDA May Not Hold Advisory Committee Meeting For DMD Therapy Approval: Stock Soars But Analyst Issues Warning
Capricor Therapeutics (CAPR) said on Tuesday that the U.S. Food and Drug Administration is not eyeing an advisory committee ...
STAT3d
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 ...
Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
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.
Second patient death reported with gene therapy for muscular dystrophy
Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...
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 ...