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FDA Approves Sarepta Duchenne Drug in Stunning Reversal

December 14, 2019, 12:03 AM UTC

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Happy Friday, readers.

In 2016, biotech Sarepta Therapeutics made waves when the Food and Drug Administration (FDA) rebuked its own scientific advisers’ opinions and approved the firm’s pioneering drug for Duchenne muscular dystrophy, a rare muscle-wasting disorder that can leave patients dead by their mid-20s.

It was déjà vu all over again this week as the agency green lit Sarepta’s Vyondys 53—less than six months after declining an approval based on toxicity and safety concerns. Sarepta stock rocketed up more than 31% on Friday following the decision.

This is a complicated and personal issue that has, at times, pitted patient advocates and members of the scientific community against each other.

For some context: Exondys 51, the first Sarepta muscular dystrophy drug cleared by the FDA, was highly polarizing. An independent panel voted against recommending the drug’s approval in a 7-6 vote back in 2016—but a concerted campaign by certain patient groups appeared to have shifted the power dynamic and led to the drug’s eventual approval.

According to Reuters, the FDA says that it had “considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease, and the lack of available therapy” in its decision to approve Vyondys 53.

It goes to show how difficult it can be to balance the hopes of patient communities with the FDA’s standards. The regulators have a dual mandate of balancing safety and efficacy. But a well-designed patient movement can sway what happens, too—and that could have far-reaching implications for plenty of other treatments.

Read on for the day’s news, and have a wonderful weekend.

Sy Mukherjee
sayak.mukherjee@fortune.com
@the_sy_guy

DIGITAL HEALTH

Apple, Color to offer free genetic tests to Silicon Valley employees. CNBC's Chrissy Farr reports that Apple, in a partnership with Color Genomics, will be offering up free genetic screenings at on-site health clinics in Silicon Valley. It's the latest in a series of moves associated with Apple's new "AC Wellness" clinics for employees—and another example of a tech giant making moves into the health care space. But the bigger question is how such efforts will fare. Are they pilot projects for even bigger plans coming down the pike? Will they actually do anything to reduce health care costs or improve outcomes (even within Apple's own employee base)? (CNBC)

INDICATIONS

Amarin scores expanded FDA approval for its fish oil drug to prevent heart attacks and stroke. Some breaking news here (the FDA seems to be on a mission to approve or expand the labels of as many drugs as possible before the end of the year): Amarin stock is on the rise after the agency endorsed its prescription strength fish oil pill Vascepa as a treatment that can reduce the risk of cardiovascular catastrophes such as heart attack and stroke among certain patients with high levels of triglycerides. "Vascepa is the first FDA approved drug to reduce cardiovascular risk among patients with elevated triglyceride levels as an add-on to maximally tolerated statin therapy," according to the FDA.

THE BIG PICTURE

Vaping deaths rise to 52, with 2,409 hospitalizations. The latest Centers for Disease Control (CDC) update on vaping-related deaths finds that 52 people have died from lung illnesses and more than 2,400 have been hospitalized. As we've reported previously, marijuana-vaping pods, and the compound Vitamin E acetate, appear to be central to this outbreak. (Reuters)

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