FDA Gene Therapy Concerns

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FDA, Gene Therapy and Sarepta

FDA’s Prasad Exits After Sarepta Gene Therapy Controversy

Vinay Prasad, a top regulator at the US Food and Drug Administration, has left the agency after a controversy over his handling of Sarepta Therapeutics Inc.’s gene therapy.

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FDA opens probe into mysterious death linked to gene therapy

Fierce Pharma · 1d

FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients

· 18h

Sarepta shares rebound after shipments of gene therapy Elevidys resume in US

Sarepta Therapeutics (SRPT.O) shares surged more than 30% before the bell on Tuesday, as analysts said the resumption of U.S. shipments for its muscular gene therapy partially removes financial headw...

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FDA Probes Death Of 8-Year-Old Who Received Sarepta's Elevidys

· 1d

Sarepta resumes shipping of gene therapy Elevidys to patients who can walk

Just The News2h

FDA allows distribution of muscular dystrophy drug again after public criticism

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy raised questions about a key FDA official who broke with Trump on regulation.

Fierce Biotech8d

Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy

The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb-girdle muscular dystrophy (LGMD) trials, while also revoking the biopharma’s gene therapy platform technology designation.

PharmExec6d

Recapping the Sarepta Saga—And Implications for Industry

A timeline of key developments surrounding Sarepta Therapeutics' Duchenne muscular dystrophy drug Elevidys, and the potential future impacts for gene therapy regulation and market access.

pharmaphorum18h

FDA gives the go-ahead to restart Elevidys shipments

In a statement, the Cambridge, Massachusetts-based company said that shipments of Elevidys (delandistrogene moxeparvovec) will restart while it continues dialogue with the FDA "on next steps in the safety labelling process and risk-mitigation approach for non-ambulatory patients."

Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys

FDA places clinical hold on Sarepta's LGMD gene therapy trials after three deaths and revokes platform tech designation due to safety concerns.