Douglas Ingram, CEO of Sarepta Therapeutics, faced challenges at the Forbes Healthcare Summit as the company’s stock plummeted over 30% due to regulatory issues with its gene therapy, Elevidys. The FDA has asked Sarepta to suspend treatment delivery while investigating patient deaths linked to Elevidys and another experimental therapy. Despite conditional approval for specific age groups, concerns persist about the therapy’s efficacy for more advanced stages of Duchenne muscular dystrophy (DMD).
Although Elevidys failed to meet some clinical trial benchmarks, Sarepta maintains that it shows promise. Investor anxiety is high, particularly regarding the drug’s market status, which is crucial for the company’s finances.
Families affected by DMD, such as Jennifer Hand, shared their experiences, highlighting the trepidation and necessity of weighing risks against potential benefits. Overall, the future of Elevidys and Sarepta’s market position remains uncertain amid regulatory scrutiny.
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