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FDA Reverses Course, Allows Sarepta’s Duchenne Gene Therapy for Younger Patients

FDA Reverses Course, Allows Sarepta’s Duchenne Gene Therapy for Younger Patients

FDA Reverses Course, Allows Sarepta’s Duchenne Gene Therapy for Younger Patients In a significant and swift reversal, the U.S. Food and Drug Administration (FDA) announced on Monday that it has cleared Sarepta Therapeutics to resume shipments of its gene therapy, Elevidys, for Duchenne muscular dystrophy. The decision specifically applies to younger Duchenne patients who retain the ability to walk. This move comes after a brief, voluntary hold on the therapy’s distribution. The FDA stated it would continue to collaborate with…

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FDA Official Signals Tough Road for Sarepta’s Shelved Duchenne Therapy Elevidys

FDA Official Signals Tough Road for Sarepta’s Shelved Duchenne Therapy Elevidys

FDA Official Signals Tough Road for Sarepta’s Shelved Duchenne Therapy Elevidys Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, Elevidys, faces a significant challenge to return to the market, according to a senior Food and Drug Administration official. The therapy was temporarily pulled due to severe safety concerns, including liver injuries linked to the deaths of two patients. Speaking anonymously to STAT, the FDA official described the path for Elevidys’ reinstatement as “arduous and treacherous,” indicating that the therapy’s license…

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Sarepta Defies FDA Order to Halt Gene Therapy Shipments Amid Rising Patient Deaths

Sarepta Defies FDA Order to Halt Gene Therapy Shipments Amid Rising Patient Deaths

Sarepta Defies FDA Order to Halt Gene Therapy Shipments Amid Rising Patient Deaths Sarepta Therapeutics has publicly defied a rare request from the U.S. Food and Drug Administration (FDA) to immediately suspend all shipments of its gene therapy, Elevidys, following the confirmed death of a third patient. The biopharmaceutical company’s refusal marks a highly unusual standoff with the federal agency, intensifying scrutiny on its leading Duchenne muscular dystrophy treatment. The FDA disclosed late Friday that it had met with Sarepta…

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