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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.


Pharmalot: You told Wall Street analysts on a teleconference that the company “tried to be reasonable” about the $300,000 annual net cost. How so? And what kind of reimbursement are you expecting?

Kaye: We fell right in the middle of the rare disease pricing, and, yes, we tried to be as thoughtful as we could. We want to make sure that patients do get the drug and get reimbursed. The price reflects our cost. This includes manufacturing, and we’ll be spending a lot of resources and effort to reinvest in Duchenne drug and get them approved for other patient populations. And there are post-marketing requirements [from the FDA to run additional studies]. So we’re spending a lot of dollars on research really for the next decade. This is not inexpensive.


And payers are paying for innovation. We’re not trying to be like Valeant, where you take a drug that’s been around for years and then raise the price. What we’ve heard in discussions [with payers] is that we’re below the point where people would become concerned. We’ve tried to be very careful, and we’re confident we will get reimbursed for this drug.

Pharmalot: You’ve not said how many boys may be eligible for treatment [different gene mutations mean the Sarepta drug is only suitable for some boys with Duchenne]. So the revenue potential is unclear, but how big is the overall market?

Kaye: We know the overall estimate is from 1 in 3,500 to 1 in 5,000 boys are born with this disease. … But there will likely be newborn screening [to identify children] that may be mandated by different states. It’s not mandated right now, but there is a presidential advisory committee reviewing this and so it could happen in the next few years. … But there has to be a recommendation and then a requirement has to be made by each state.

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