The Sarepta saga never ends
Last year, Sarepta Therapeutics went round-trip through the FDA with its latest treatment for Duchenne muscular dystrophy, getting rejected in August before successfully appealing the decision in December. Yesterday, we got a glimpse at the FDA’s internal deliberations on the matter, and, as one might expect, they provided ample fodder for fighting over how drugs get approved in the 21st century.
As STAT’s Matthew Herper reports, the initial rejection was based on scant evidence that Sarepta’s drug, Vyondys 53, could provide enough benefit to patients with Duchenne to overcome its side effects. But the story, as is often the case with Sarepta, is more complicated than that.
The FDA’s concerns date back to 2016, when similar infighting led to the approval of Sarepta’s first Duchenne treatment, Exondys 51, also granted with little in the way of concrete proof. To dissenting FDA staff, the Exondys experience was reason to reject Vyondys; to the regulators who overruled that decision, it was a precedent that made Vyondys approvable. And to some outside critics, the whole experience is cause for concern that FDA standards are eroding before our eyes.
Read more.
As STAT’s Matthew Herper reports, the initial rejection was based on scant evidence that Sarepta’s drug, Vyondys 53, could provide enough benefit to patients with Duchenne to overcome its side effects. But the story, as is often the case with Sarepta, is more complicated than that.
The FDA’s concerns date back to 2016, when similar infighting led to the approval of Sarepta’s first Duchenne treatment, Exondys 51, also granted with little in the way of concrete proof. To dissenting FDA staff, the Exondys experience was reason to reject Vyondys; to the regulators who overruled that decision, it was a precedent that made Vyondys approvable. And to some outside critics, the whole experience is cause for concern that FDA standards are eroding before our eyes.
Read more.
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