Sarepta Therapeutics is facing scrutiny after a patient died while receiving Elevidys, its gene therapy for Duchenne muscular dystrophy. Although acute liver injury is a known side effect, the severity of this case was unprecedented. Elevidys, which received expedited U.S. approval amid efficacy concerns, has been administered to over 800 patients. The FDA's prior rapid endorsements of Sarepta's treatments raise ethical questions about their effectiveness, especially as none of their other drugs have confirmed benefits thus far. Following the incident, Sarepta will revise Elevidys' prescribing information and the company's share price dropped significantly by over 23%.
The tragic death of a patient on Sarepta's Elevidys highlights the urgent need for continued monitoring and research into the long-term safety of gene therapies.
Sarepta plans to update Elevidys' prescribing information after a patient's acute liver injury, prompting concerns over the gene therapy's risks and benefits.
Despite the approval of Elevidys by the FDA, skepticism remains about its efficacy in treating Duchenne muscular dystrophy, raising questions about the trajectory of its future.
This occurrence of a death linked to Elevidys, the first reported in over 800 patients treated, emphasizes the unpredictable nature of gene therapy outcomes.
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