June 28, 2024

The need-to-know this morning

The FDA rejected Rocket Pharma's gene therapy for severe leukocyte adhesion deficiency-I, a rare, sometimes fatal immunodeficiency syndrome. The company said the agency had manufacturing concerns.
Apellis said CHMP, the drug-reviewing arm of the European Medicines Agency, recommended against approving Empaveli, its injectable for geographic atrophy. Apellis said it plans to appeal.
CHMP also recommended against approving an ALS drug and once again recommended the EMA not renew authorization for Translarna, a controversial Duchenne muscular dystrophy drug.
A tiny Swiss biotech's long Covid trial failed, triggering layoffs.

legislation

House committee passes four health bills

The House Ways & Means Committee just passed four important federal health reimbursement bills. Among them was new legislation that would expand Medicare coverage of obesity drugs, which until now has been prohibited. The bill would only cover certain of obesity patients, however — paying for patients who were taking weight loss drugs before transitioning to Medicare.

A separate bill would allow Medicare to pay for healthy people to take cancer screening blood tests. Galleri, a multi-cancer test, has a list price of about $950; the bill would allow Medicare to pay $508 for such tests.

The committee also voted in favor of a bill centered on Medicare coverage of breakthrough medical devices, and unanimously passed another that would force Medicare to reconsider special coverage decisions after 10 years.

psychedelics

Why MDMA-assisted therapy still might win approval

Despite an advisory committee overwhelmingly rejecting a bid from Lykos Therapeutics to use MDMA as a treatment for PTSD, founder Rick Doblin has remained optimistic. At a recent psychedelics conference, he said he believed there was "substantially more than a 50/50 chance" that the FDA would approve the psychedelic.

Doblin and other psychedelics evangelists have been working on persuading regulators since the 1980s that this treatment modality is effective. The decades of work could ultimately pay off — and Lykos very well may see an approval this summer for its MDMA-based treatment, STAT's Olivia Goldhill writes.

podcast

The important health issues this election season

Will Medicare finally begin covering weight loss drugs like Zepbound and Wegovy? And what health care issues may be lightening rods in this election?

STAT chief Washington correspondent Rachel Cohrs Zhang joins the podcast to talk about a new proposal for obesity drug coverage in Washington and what to expect from the first presidential debate. STAT health tech reporter Brittany Trang also guest co-hosts "The Readout LOUD" this week, where we discuss AI for drug development, the latest in the H5N1 bird flu outbreak, a surprise entrant to the GLP-1 field, and more news in the life sciences.

Listen here.

biosecurity

Controlling those biotech supply chains

America's ability to stay on top of the biotech industry will depend on its ability to protect genomic data and intellectual property, opines Josh Carpenter, CEO of a nonprofit translational research institute focused on drug discovery and genomic research. These are the critical tools in developing more advanced precision medicine in an increasingly complex international market.

"China poses a threat to both securing genomic data and protecting intellectual property," he writes. "This misappropriation of genomic data and fecklessness with IP underscores the need for stringent protections from entities that pose such risks."