July 25, 2025

The need-to-know this morning

European regulators said Sarepta Therapeutics' Duchenne muscular dystrophy gene therapy should not be approved, further imperiling the future of the treatment, which is also facing increasing scrutiny in the U.S. Roche has commercial rights to the gene therapy, called Elevidys, in Europe and other countries.
European regulators said Eli Lilly’s Alzheimer’s treatment Kisunla should be approved for a select group of patients, changing course from an initial recommendation to reject the drug.
Bristol Myers Squibb named Cristian Massacesi as its new chief medical officer. Massacesi is a phyisican and most recently served as chief medical officer at AstraZeneca and its Alexion unit. He replaces Samit Hirawat, who served as Bristol's CMO for the past six years.

RARE DISEASE

Families devastated after FDA halts Sarepta gene therapy

Families of boys with Duchenne muscular dystrophy were devastated when the Food and Drug Administration asked Sarepta Therapeutics to halt shipments of its gene therapy, Elevidys, citing safety concerns — and the company subsequently agreed earlier this week. Parents who had fought for months to secure access are now stuck in limbo, with appointments abruptly canceled and little communication from regulators, STAT's Jason Mast reports.

The emotional whiplash has been severe — particularly for the kids who had already been prepped for the $3.2 million infusion. One child, Hunter, was ready to be dosed next week — and he was hopeful that the therapy might help him run or walk up stairs a bit more like his classmates could. His mother has started a Change.org petition to try and allow the drug back on the market, but in the meantime, she still hasn’t sat down with Hunter.

“We don’t know how to tell him,” she told Jason.

animal testing

Federal move away from animal testing draws mixed reactions

The Trump administration is aggressively pushing federal agencies including the NIH and FDA to replace animal testing with so-called new approach methodologies — AI, organoids, organ-on-a chip tech, and other such tools. The NIH will no longer fund proposals exclusively reliant on animals.

The shift is being lauded as both ethically overdue and technologically forward-thinking — or, conversely, naive and politically motivated, STAT’s Marissa Russo and Jonathan Wosen report.

“There's a lot of enthusiasm, but if that enthusiasm pushes things forward too quickly and outpaces the careful validation that is really needed, then we run an ethical risk that you're going to advance things to human trials that could put patients at risk,” one expert said.

depression

FDA panel stirs backlash for SSRI use during pregnancy

The FDA earlier this week convened a panel on antidepressant use during pregnancy that relied heavily on the opinions of SSRI skeptics, while sidelining other maternal health experts. Despite a robust body of evidence showing that untreated depression poses severe risks, much of the discussion focused on the notion that antidepressants are harmful to developing babies, a suggestion that is not supported by the weight of medical evidence.

“The absolute risk of birth defects is very small,” one birth defects epidemiologist at the University of British Columbia told STAT’s Lizzy Lawrence. “What's dangerous is creating fear that leads people to stop treatment abruptly or avoid care entirely.”

podcast

A mother's perspective on Sarepta's gene therapy halt

How are families of patients reacting to the removal of Sarepta's gene therapy from market? Why are investors excited about narcolpesy drugs? And why has there been less financing in oncology startups?

We chat about all that and more on this on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. We bring on a mother whose 7-year-old has Duchenne muscular dystrophy, and hear her thoughts on Sarepta's decision to pull its treatment Elevidys from the market, following a request from the Food and Drug Administration.

We also talk about a rare mega-round for a new oncology biotech and an emerging class of drugs aimed at helping people stay awake.

Listen here.

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