FDA Sarepta documents raise questions about fast drug approvals

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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

INDICATIONS

A mixed earnings report for J&J—but good times could be around the corner, according to one analyst. Johnson & Johnson shares fell slightly on Wednesday following a mixed earnings report that fell short of Wall Street expectations on profits and revenues. The company has been facing competition from generic rivals from its massive pharmaceuticals unit. But SVB Leerink's Danielle Antalffy writes in an investor note that shares are likely to move higher over the course of 2020, especially as the firm's various litigation costs (including in its class action talc powder cases) continue to subside.

THE BIG PICTURE

China escalates its response to the coronavirus outbreak. The coronavirus outbreak—which has reached U.S. shores, according to the CDC, after a Washington man returned from a trip to China—has prompted Chinese authorities to shut down major modes of transportation from Wuhan, a city inhabited by more than 11 million people and the seeming epicenter of the respiratory disease-causing virus. A man in Mexico is also reportedly being examined for possible infection by this coronavirus strain (which is different from others related to conditions like SARS). (New York Times)