One the FDA's biggest controversies is coming back to test its new leadership.
Sarepta Therapeutics Inc. on Wednesday released promising early data from a study of a new drug to treat some patients with the rare muscle-wasting disease Duchenne Muscular Dystrophy (DMD). The drug is a follow-up to Exondys 51, another DMD treatment the FDA approved last year -- over the objection of some of its own scientists -- to treat a different subset of patients. The company plans to ask the FDA if it can seek accelerated approval of the medicine, according to Leerink analyst Joseph Schwartz. Its shares rose 11 percent on Wednesday.
No company has benefited more than Sarepta from an FDA shift in recent years toward greater flexibility in approving drugs for rare and deadly conditions such as DMD. And the company may reap more rewards from a new FDA regime that appears open to continuing that shift.
The FDA drug-approval process can be on the dry side -- heavy on PowerPoint and side-effect analysis. The saga of Exondys's approval was operatic in comparison.
Sarepta sought approval for that drug based on a tiny study of just 12 boys that wasn't blinded or placebo-controlled. The medicine caused a civil war at the FDA, between scientists who believed Sarepta hadn't proved its drug worked and an administrator worried that requiring another study would cause a cash crunch at Sarepta and harm patients with a deadly disease and no options. The fact that Exondys was approved under such circumstances suggested the FDA's approval bar was flexible.
There's evidence the man running the FDA since May, Commissioner Scott Gottlieb, may be extra bendy.
His FDA has recently reversed course in favor of an Eli Lilly & Co. arthritis drug and an Amicus Therapeutics Inc. rare-disease drug. Neither company will have to run additional, lengthy clinical trials the FDA had previously demanded.
Gottlieb's FDA has approved several drugs ahead of schedule, including the first of a new generation of cancer therapies that modify human immune cells to potentially wipe out cancers. Already, 2017 is the fourth most-productive year for drug approvals since 2005. Gottlieb also made comments during congressional testimony earlier this year that suggested he supports more FDA flexibility on clinical trial design.
Sarepta may need this extra flexibility if it wants rapid approval of its second DMD medicine. Wednesday's release is an early look at another tiny study. And there is no proof yet the drug provides direct patient benefit -- only that it increases levels of a protein DMD patients need, and it's a very small increase.
Gottlieb's predecessor at the FDA, Robert Califf, went out of his way to say Exondys's initial approval shouldn't set a precedent. The FDA made its approval conditional on Sarepta running another study to confirm Exondys works; that hasn't happened yet.
But the results Sarepta released Wednesday suggest its new medicine is potentially more effective than Exondys at raising that protein level. It may be hard for the FDA to say this isn't good enough for a possible accelerated approval, after already giving such treatment to Exondys.
If the FDA lets Sarepta apply for accelerated approval, then the new drug could come to market quite rapidly. That would be a big deal in terms of increasing the number of boys Sarepta's treatments can reach -- and an even bigger validation of the company's pipeline and strategy. Otherwise, it may be a long wait for a second drug.
The FDA's handling of Sarepta's new drug could also possibly be the strongest signal yet of what the industry can expect from Gottlieb's tenure.
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