Does the FDA need to slow its drug approval roll?

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Happy Wednesday, readers.

Sarepta Therapeutics has a way of bringing out the passions in the biotech world.

Its pioneering treatments for Duchenne muscular dystrophy—Exondys 51, approved in 2016, and Vyondys 53, cleared by the Food and Drug Administration (FDA) in late 2019—were either marquee victories for patients suffering from a devastating muscle-wasting rare disease or an abrogation of the FDA’s duty to judge medicines solely on their safety and efficacy, depending on your perspective. (Read these two pieces for some more background on the controversy.)

Sarepta was back in the news Wednesday following newly published FDA documents highlighting considerable disagreement amongst the agency’s own expert staff on whether or not Vyondys 53 should be approved despite safety and efficacy concerns.

It’s a rare look into the FDA’s drug approval process and communications with companies (these sorts of communications, called Complete Response Letters, are almost never made public unless the therapy in question actually is approved)—and it raises the larger question of whether or not the agency has been pushing new treatments across the finish line with a bit too much zeal.

In 2019, the FDA approved 48 new drugs. The number was 59 in 2018. Those are massive figures in the drug approval world, especially compared with the previous 10 years. And the pace of drug green lights aligns with a deliberate undertaking by current (and recent) FDA leaders to shepherd new therapies to the market.

But here’s the rub. These accelerated approvals, often pushed by patient groups which argue that something is better than nothing when it comes to life-threatening rare disorders, require post-market surveillance for more data. In other words: The clinical trial may play out in actual patients in the real world before a drug’s safety and efficacy has been definitively proven. And, in some cases, the “confirmatory” trial for these drugs are never actually completed, according to a study by Yale, NYU, and Dartmouth researchers.

That’s an approach that has its fair share of proponents given how devastating some of these diseases can be. But the internal politicking at the FDA demonstrated in the newly released documents shows how tricky the balance can be.

Read on for the day’s news.

Sy Mukherjee
sayak.mukherjee@fortune.com
@the_sy_guy

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