The need-to-know this morning

Roche said it will run another trial of Elevidys, the Duchenne muscular dystrophy gene therapy developed by Sarepta Therapeutics, in an attempt to win European approval. Sarepta markets the drug in the U.S., while Roche has rights to the therapy outside the U.S.
Eli Lilly said its GLP-1 pill Foundayo showed non-inferiority compared with insulin glargine in a cardiovascular outcomes trial of people with diabetes and obesity. The company said it will submit Foundayo to the FDA as a treatment for type 2 diabetes by the end of the second quarter under the Commissioner's National Priority Review Voucher.

WASHINGTON

FDA reopens door to compounded peptides

The Food and Drug Administration is calling for an outside advisory panel to reconsider whether compounding pharmacies should be allowed to make a spate of controversial peptides it previously moved to restrict. The first meetings will be in July, and a later one will be held before February 2027, STAT’s Lizzy Lawrence and Sarah Todd write.

Many of these compounds — hyped online for everything from muscle growth to anti-aging — have little credible safety or efficacy data, experts say. And there have been some concerning signals like tumor growth or immune reactions. Still, peptides are gaining traction with influencers and backing from HHS Secretary Robert F. Kennedy Jr., who argues that regulated peptide compounding could pull demand away from the black market.

“I just hope the committee does the right thing and [does] not lift the ban on these peptides for compounding in the U.S. given their vacuous data for safety and efficacy,” Eric Topol, a longevity expert and director and founder of Scripps Research Translational Institute, told STAT via email.

Alzheimer's disease

Cochrane review reignites Alzheimer's amyloid wars

A new Cochrane meta-analysis of 17 trials and more than 20,000 patients determined that anti-amyloid drugs “show no clinically meaningful benefit.” But the analysis hinges on pooling data across a broad mix of amyloid-targeting programs — including failed drugs — effectively treating the class as uniform despite differences in mechanism, potency, and clinical performance.

The Cochrane review reinforced the contentious idea that clearing amyloid plaques doesn’t translate into meaningful patient benefit, even though it can hit individual biomarkers. The concern is that the meta-analysis could actually obscure positive signals from individual therapies that have shown better results in early stage disease.

The Alzheimer’s Association pushed back, saying the “analysis inappropriately includes a more broad, less relevant range of studies. Glaringly missing from this analysis is that behind every data point is a person.”

REGULATION

Did money buy a drug approval?

The FDA last week approved Travere Therapeutics' Filspari for a rare kidney disease, even though the drug failed its main clinical goal, preserving kidney function, STAT’s Adam Feuerstein writes in his weekly Biotech Scorecard newsletter. Actually, it performed slightly worse than the control.

What changed wasn’t the data, but the endpoint: After initially rejecting the drug, the agency pivoted to accept proteinuria reduction as a surrogate marker. This was in part due to a conveniently timed, industry-linked effort that helped validate it.

That shift turned a losing trial into a regulatory win, giving Filspari the first-ever approval in FSGS and sending Travere’s stock soaring — even though the label showed the drug didn’t meet its primary endpoint. Adam writes that it's a striking example of how flexible — or malleable — FDA standards can be when the right scientific and political pieces fall into place.

DEVICES

Medicare tightens standards for breakthrough device payments

Medicare plans to roll back an easier payment pathway for FDA-designated breakthrough devices, STAT’s Katie Palmer writes. It argues that being expensive simply isn’t enough to justify extra reimbursement if there isn’t clear evidence of clinical benefit.

A proposed rule would require all devices — including breakthroughs — to prove they’re both new and meaningfully better than existing options to qualify for add-on payment.

“This challenges the idea that we could presume a device is providing substantial clinical improvement compared to existing technologies, when there is no clinical evidence even used to justify the authorization in the first place,” said Kushal Kadakia, a resident at Massachusetts General Hospital who studies medical device regulation.