After months of controversy, the Food and Drug Administration on Monday approved a Sarepta Therapeutics drug for Duchenne muscular dystrophy, a rare disease that confines boys to wheelchairs and sends them to an early death. But debate continues over whether the FDA lowered standards to approve a drug for an unmet medical need. And the price is a concern, too. The drug will cost about $300,000 per patient per year, depending upon weight, which means the price can run much higher. We spoke briefly with Dr. Ed Kaye, the interim Sarepta chief executive, about the approval. This is an edited version of the conversation.
Pharmalot: You managed to navigate a difficult process, but if critics are right, why shouldn’t we all be concerned that, perhaps, the FDA did not adhere to approval standards? There may be potential implications for anyone who takes new medicines down the road.
Kaye: We’ve pursued the approval process by using a surrogate endpoint [a measure suggesting a clinical benefit] and that’s very well established in the law. We were able on different occasions to demonstrate that [increasing a protein called] dystropin reached the endpoint. … Now we have to show a clinical benefit. That’s the challenge we have … I think [reliance on surrogate endpoints] is probably to become more common. Remember that Congress wrote legislation to make sure that we get that done. So it’s a learning process for us and the FDA. It was a little bit controversial. … But I think there needs to be some changes, because what we’re talking about here is really personalized medicine. We’re pushing the science, but also have to push the regulatory science as we go along.
California already has 57 MANDATED newborn genetic screening tests. These diseases are single gene mutations with 100% positive predictive value for those afflicted. The anomalous gene mutations in DMD that influence treatment outcomes may complicate the development of a newborn screening with significant predictive value.
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