FDA Approves Sarepta Duchenne Drug in Stunning Reversal

December 14, 2019, 12:03 AM UTC

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Happy Friday, readers.

In 2016, biotech Sarepta Therapeutics made waves when the Food and Drug Administration (FDA) rebuked its own scientific advisers’ opinions and approved the firm’s pioneering drug for Duchenne muscular dystrophy, a rare muscle-wasting disorder that can leave patients dead by their mid-20s.

It was déjà vu all over again this week as the agency green lit Sarepta’s Vyondys 53—less than six months after declining an approval based on toxicity and safety concerns. Sarepta stock rocketed up more than 31% on Friday following the decision.

This is a complicated and personal issue that has, at times, pitted patient advocates and members of the scientific community against each other.

For some context: Exondys 51, the first Sarepta muscular dystrophy drug cleared by the FDA, was highly polarizing. An independent panel voted against recommending the drug’s approval in a 7-6 vote back in 2016—but a concerted campaign by certain patient groups appeared to have shifted the power dynamic and led to the drug’s eventual approval.

According to Reuters, the FDA says that it had “considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease, and the lack of available therapy” in its decision to approve Vyondys 53.

It goes to show how difficult it can be to balance the hopes of patient communities with the FDA’s standards. The regulators have a dual mandate of balancing safety and efficacy. But a well-designed patient movement can sway what happens, too—and that could have far-reaching implications for plenty of other treatments.

Read on for the day’s news, and have a wonderful weekend.

Sy Mukherjee


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