Makary goes off script on rare disease

FDA Commissioner Marty Makary went on CNBC yesterday to answer a host of questions, and in the process defended a string of rare disease drug rejections. He included what sounded like a pointed reference to UniQure's Huntington's gene therapy, STAT's Lizzy Lawrence and John Wilkerson write, describing a trial involving burr holes drilled into patients' skulls that showed "no benefit" despite pressure to approve. It's highly unusual for a sitting commissioner to publicly discuss the fate of specific products under review, and UniQure's stock fell over 30%.

Makary also forcefully lauded CBER chief Vinay Prasad as a "genius," dismissing criticism as a "fatwa" that's been "put on Vinay Prasad by one or two media outlets."

No fatwas have been issued by STAT.

fda

FDA to reward staff for speeding up drug reviews

From STAT's Lizzy Lawrence: The Food and Drug Administration will offer quarterly bonuses to staff who complete drug reviews earlier than expected. FDA Commissioner Marty Makary and deputy drug center director Mike Davis outlined the program in a town hall on Thursday.

"My job as your commissioner is to be your advocate and to fight for you," Makary said, according to a recording obtained by STAT. "It did take some wrangling, it took some not taking no for an answer, but we were able to get this done."

Davis noted that reviewers will also be judged on the quality of their work, to avoid incentivizing staff to rush through reviews.

The pilot program will start April 1 and will only apply to reviewers in the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research. Bloomberg first reported the news.

The morale of FDA employees has plummeted under the Trump administration after a series of mass layoffs, leadership turnover, and increasing political pressure on scientific activities. The bonus program seems to be an attempt to boost morale and follow through on Makary's push to speed up drug reviews.

lung cancer

Boehringer wins lung cancer approval in 44 days

Boehringer Ingelheim's HER2-mutant lung cancer drug Hernexeos just snagged its first FDA approval in just 44 days after submission — marking it the second experimental therapy cleared under Commissioner Marty Makary's new National Priority Voucher Pilot Program. The expansion moves the drug from previously treated patients into newly diagnosed metastatic non-squamous NSCLC, backed by a 76% response rate in previously untreated patients.

The win also gives Boehringer a step ahead of Bayer's rival HER2 drug, FiercePharma notes, as both race for dominance in a small but competitive slice of lung cancer — though confirmatory data will still be required.

opinion

A 'third bridge' for kids with rare disease

The FDA's draft "plausible mechanism" framework could finally create a sustainable path for individualized genetic therapies, opines Judy Stecker, a former HHS public affairs chief and rare disease mom. Writing about her 6-year-old son Wheeler, who has CLN3 Batten disease, Stecker describes a system long split between billion-dollar drug development trajectories and one-off, parent-built rope bridges like those that delivered bespoke treatments to children like Mila Makovec and Baby KJ.

The proposed framework, she says, would function as a "third bridge," allowing platform-based gene or molecular therapies to win an initial rigorous approval and then be adapted for future patients with different mutations under that same umbrella. This could potentially accelerate access without lowering standards, she writes.

The guidance is still in draft form and raises unanswered questions around manufacturing, data sharing, and implementation. But Stecker frames it as a meaningful structural shift for families who have watched scientific possibility outrun regulatory practicality.

podcast

FDA turmoil, election intrigue, AI, and more

On "The Readout LOUD" this week: Adam Feuerstein, solo. His usual co-hosts Allison DeAngelis and Elaine Chen took some time off, so Adam manned the podcast mic himself.

Adam thought, quite understandably, that our cherished listeners wouldn't want to hear him drone on for 30 minutes, so he thankfully found some help. Jared Holz, health care sector specialist at Mizuho Securities, agreed to be Adam's podcasting wingman for this week's show.

They ran through a menu of takes on biotech stock performance, M&A, Food and Drug Administration turmoil, election intrigue, and artificial intelligence.

Listen here.

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