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The Readout Damian Garde

Sarepta's once-rejected drug gets a second FDA opinion

In August, the FDA rejected Sarepta Therapeutics’ second Duchenne muscular dystrophy drug for reasons that were more than a little unclear. Last night, the agency changed its mind entirely and offered little in the way of explanation.

The FDA approved Vyondys 53 to treat the roughly 8% of Duchenne patients whose disease results from a specific genetic error. The agency’s statement made no mention of the prior rejection, but Sarepta said in a news release that it had filed an appeal with the FDA and worked to address regulators’ concerns.

As for cost, Sarepta said it will price Vyondys 53 “at parity” with its previous Duchenne treatment, Exondys 51, which is dosed based upon patients’ weight and can cost upward of $1 million per year.

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That drug pricing bill passed. Now what?

Yesterday, the House passed a bill that would cut consumer drug spending by 55% and cost the biopharma industry about $1 trillion in revenue over a decade. No one expects it to get traction in the Senate, and President Trump has promised a veto even if it did. But that doesn’t make this meaningless.

As STAT’s Lev Facher reports, the bill sets the agenda for Democrats in what is likely to be a long fight over drug prices. There’s bipartisan concern over the rising cost of medicine, and the House bill, however doomed, could serve as a stake in the ground for future negotiations.

Then there’s the upcoming election. Democrats are hoping to seize control of the White House and Senate in 2020, and the House pricing bill could double as a campaign pitch and blueprint for future legislation.

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Did the FDA forever change in 2016?

Has CAR-T lost its luster? And why is colorectal cancer on the rise? 

We discuss all that and more on the latest episode of “The Readout LOUD,” STAT’s biotech podcast. First, we dig into the news out of this week's big hematology conference, where novel treatments for blood cancer threatened the future of genetically engineered CAR-T therapies. Then, former Sarepta Therapeutics CEO joins us to talk about the company's regulatory legacy and what he's been up to since leaving it. Finally, we talk to Manju George, a patient activist working to spread the word about the alarming increase of colorectal cancer among young people.

You can listen to the episode here. To listen to future episodes, be sure to sign up on Apple Podcasts, Stitcher, Spotify, or wherever you get your podcasts.

Senate confirms Hahn as FDA commissioner

There is once more a Senate-confirmed FDA commissioner, in the form of Dr. Stephen Hahn, who got the job through a 72-18 vote yesterday.

As STAT’s Nicholas Florko reports, the MD Anderson oncologist went through with a larger margin than Dr. Scott Gottlieb, who came in with deep industry ties and won confirmation 57-42. But each is a departure from uncontentious commissioner appointments past. Dr. Robert Califf was approved 89-4, and Dr. Margaret Hamburg was confirmed unanimously.

In Hahn’s case, the concerns stem not from his stated positions but from a lack thereof. At a confirmation hearing last month, Hahn was vague on the topic of regulating e-cigarettes and provided few hints on his vision of the FDA’s future. It also remains to be seen whether he’ll unlock what appears to be his Twitter account.

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A new idea in Alzheimer’s

Earlier this year, scientists at Washington University ferreted out a genetic mutation that seemed to protect people from developing Alzheimer’s disease. What if there were a drug, they wondered, that could replicate that effect in patients at risk?

Alector, an early-stage biotech company, believes it may have found such a thing. The company’s latest drug candidate, AL014, is designed to mimic the beneficial effects of that variant, which is called MS4A4A. The idea is to empower immune cells called microglia, which clear the brain of unwanted toxins and, in theory, can be harnessed to delay the onset of Alzheimer’s.

It’s early days for AL014. Next year, Alector plans to begin the preclinical studies that would enable later human trials. But the drug, and the MS4A4A target, are among the small but growing number of new ideas in Alzheimer’s research, which has for years focused on beta-amyloid plaques with, to be kind, disappointing results.

More reads

  • U.S. Patent Office removes lawyer from high-profile case involving a Gilead HIV medicine. (STAT Plus)
  • Kite Pharma's ex-CEO contradicts founder as CAR-T patent trial heats up, with conflicting valuations. (Endpoints)
  • BMJ should retract flawed research paper on chronic fatigue syndrome. (STAT)

Thanks for reading! Until next week,

Friday, December 13, 2019


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