June 20, 2025

The need-to-know this morning

Advisers to the European Medicines Agency recommended the approval of Madrigal Pharmaceutical's drug to treat people with MASH, a type of fatty liver disease. The drug, called Rezdiffra, is already approved in the U.S.

REGULATION

FDA goes quiet on AI-enabled devices amid growth

While the FDA has raced to deploy internal AI tools under Commissioner Marty Makary, it has stopped updating its public list of AI- and machine learning-enabled medical devices — a key part of a transparency effort launched after a 2021 STAT investigation. STAT's Katie Palmer used public agency documents to identify at least 167 newly authorized devices in the nine months since the list was last updated, suggesting the pace of approvals continues even as public accountability stalls.

"I think it is important for the sake of transparency for people to know what AI-enabled devices are being authorized by the FDA," said Roxana Daneshjou, who studies AI/ML-aided medical devices at Stanford University. "And it allows us to see what level of risk these authorized devices may have."

Without candor around how these devices are trained, tested, or labeled, providers and patients alike are left in the dark about AI's role in clinical care — and researchers are hamstrung in efforts to monitor safety and equity.

REGULATION

Top gene therapy regulator forced out at FDA

Nicole Verdun, director of the office that reviews cell and gene therapies at the FDA, and her deputy Rachael Anatol were placed on administrative leave and escorted out of the agency earlier this week, according to a recording of a meeting obtained by STAT.

Verdun had worked closely with Peter Marks, the former head of the Center for Biologics Evaluation and Research, on establishing new paths for bringing gene therapies to market, particularly in rare diseases. Marks was forced out by the Trump administration in March for his role in regulating Covid-19 vaccines.

As STAT's Jason Mast and Adam Feuerstein report, Verdun was seen as a key figure in the center, especially after Marks' departure. At that time, the CEO of a gene therapy maker said: "We value continuity, so having Verdun stay at the FDA is really important."

hiv

Gilead CEO weighs in on a 'milestone moment'

This week's FDA approval of Gilead's Yeztugo, a twice-yearly injection to prevent HIV, was "a milestone moment in the journey to end" the epidemic, CEO Daniel O'Day told STAT's Matthew Herper in a new interview.

"It can be difficult to put into words sometimes, but I think it's hard to overstate what I believe this means for public health," O'Day said. "It's nothing short of extraordinary, and could help millions of people around the world."

The celebration is tempered by political uncertainty: The Trump administration's cuts to HIV prevention raise questions about whether the drug will reach the people who need it most.

PODCAST

FDA's gene therapy turmoil unpacked

Why did the FDA suddenly oust one of its top gene therapy regulators? Is the agency growing too political? And should private firms fund academic research?

We chat about all that and more on this on this week's episode of "The Readout LOUD," STAT's biotech podcast. We discuss the tragic news that a second teenage boy with Duchenne muscular dystrophy died after taking Sarepta Therapeutics' gene therapy, raising renewed questions about the FDA's standards for approving drugs.

Listen here.

alzheimer's disease

U.K. rejects Alzheimer's drugs over cost concerns

The U.K. health agency NICE has reaffirmed its conclusion that the Alzheimer's drugs Leqembi (made by Biogen and its partner Eisai) and Kisunla (made by Eli Lilly) are not cost-effective, meaning the medicines won't be made available through the National Health Service.

In statements, Lilly and Eisai said they planned to appeal the decisions, STAT's Andrew Joseph reports.

"We feel it is our duty to sound the alarm today that this appraisal process needs an immediate overhaul so that future medicines may be more appropriately assessed, and patients and their families can have hope regarding future treatments," said Gary Hendler, an Eisai executive.

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