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FDA Halts Sarepta's Controversial Gene Therapy After Deaths

8/11/2025

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Summary

Story summary

In a controversial move, the FDA granted accelerated approval for Sarepta Therapeutics' $3.2 million gene therapy for Duchenne muscular dystrophy in 2023, despite scant clinical evidence. The situation worsened when three patients suffered fatal liver failures, prompting the FDA to halt distribution and sending Sarepta's stock spiraling down by nearly 50%.

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Story summary

In a controversial move, the FDA granted accelerated approval for Sarepta Therapeutics' $3.2 million gene therapy for Duchenne muscular dystrophy in 2023, despite scant clinical evidence. The situation worsened when three patients suffered fatal liver failures, prompting the FDA to halt distribution and sending Sarepta's stock spiraling down by nearly 50%.