News

Sarepta soars after FDA says some patients can get Elevidys

Sarepta Therapeutics Inc. shares rose sharply early Tuesday after US regulators reversed course and recommended that patients ...
Rheumatology Advisor4h

Elevidys Use for Ambulatory DMD Patients May Resume, Says FDA

The FDA is allowing Sarepta to resume shipments of Elevidys (delandistrogene moxeparvovec) to ambulatory patients with Duchenne muscular dystrophy.
BioSpace1d

‘Unprecedented’ FDA Leaks Sow Confusion For Patients, Sarepta and Capricor

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

FDA’s Reversal on Gene Therapy Helps My Son

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

Trump drove firing of FDA official

The president overruled his HHS secretary and FDA chief, four people with knowledge of the decision tell POLITICO.
Clinical Trials Arena on MSN1d

Hansa’s Idefirix shows potential as pre-treatment to Elevidys

Hansa Biosciences’ Idefirix (imlifidase) has reduced patients’ antibodies to a level that allows dosing in patients with Duchenne muscular dystrophy (DMD) with Sarepta and Roche’s Elevidys ...
Fierce Pharma5d

Vinay Prasad's exit signals FDA's probable slip toward more permissive regulation of cell and gene therapy: analyst

But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...
PharmExec6d

Sarepta Stock On the Rise Following Resumption of Elevidys Shipments in US

While MSN reports that Sarepta’s stock price began to rise on Monday on the heels of the announcement, 5 the company is still ...
The American Journal of Managed Care5d

FDA Recommends Sarepta Resume DMD Gene Therapy Shipments for Ambulatory Patients

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

Vinay Prasad, controversial FDA official, abruptly departs agency

Prasad's exit ends a tumultuous tenure during which he led a reworking of agency guidelines on COVID vaccines and his office ...
PharmExec6d

Vinay Prasad’s FDA Departure Follows Controversy Over Drug Rejections

The FDA’s top vaccine and gene therapy official resigned amid heightened scrutiny over recent drug approval decisions and ...
Just The News6d

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.