FDA casts doubt on Capricor's Duchenne stem cell therapy
The FDA says Capricor's Duchenne stem cell therapy deramiocel did not meet the Phase 3 trial endpoints, contradicting the company’s earlier claim of success and ahead of an upcoming hearing.
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FDA Requests Halt on Sarepta's Duchenne Gene Therapy Amid Safety Concerns
The FDA is expected to request Sarepta Therapeutics to halt shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, following safety concerns and patient deaths related to liver failure, which may lead to a market withdrawal amid ongoing safety investigations.
Sarepta's Duchenne Gene Therapy Linked to Second Patient Death
Sarepta Therapeutics has halted shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, after the death of a second non-ambulatory patient due to acute liver failure, prompting the company to work with experts and the FDA to develop a safer immunosuppressive regimen.