January 30, 2026

The need-to-know this morning

Regeneron Pharmaceuticals expects an FDA approval decision in the first half of this year for its gene therapy to treat a rare, genetic cause of hearing loss. An application for the therapy, called DB-OTO, was filed in December, the company said. The FDA awarded DB-OTO a Commissioner's National Priority Voucher last October. Regeneron also reported fourth-quarter and 2025 earnings.
European regulators have started a review of Amgen's drug Tavneos following "emerging information that raises questions regarding the data integrity" of the main study that supported approval, the European Medicines Agency said. Tavneos is used to treat patients with two, severe inflammatory conditions affecting blood vessels.
Moderna Chief Medical Officer Jaqueline Miller is stepping down, the company said. No reason was given for her departure. Moderna also named David Berman as its new chief development officer. Berman was most recently the R&D chief at Immunocore, a maker of cell-based immunotherapies.

white house

TrumpRx launch is delayed, though details are murky

From STAT's John Wilkerson: The Trump administration was expected to launch its TrumpRx website today, but those plans have been delayed, according to individuals familiar with the effort. Health secretary Robert F. Kennedy Jr. said yesterday during a cabinet meeting to expect the launch sometime "in the next 10 days," while one report emerged suggesting technical problems were to blame for delays.

TrumpRx is an integral part of the administration's overall plan to lower drug prices — specifically as a tool for consumers without insurance. The government website will not sell medicines. Instead, it's supposed to refer consumers to websites where they can buy drugs directly from companies. But many details are still not known, including which brand-name drugs will be listed on the site.

Yesterday, Sens. Dick Durbin (D-Ill.), Elizabeth Warren (D-Mass.), and Peter Welch (D-Vt.) sent a letter to the Office of Inspector General at the Department of Health and Human Services raising questions about TrumpRx, saying they had concerns about "inappropriate prescribing, conflicts of interest, and inadequate care."

Deals

AstraZeneca looks to China for obesity drug candidates

From STAT's Andrew Joseph: AstraZeneca has picked up additional weight loss drug candidates, with the U.K. pharma saying this morning that it was now partnering with China's CSPC Pharmaceuticals across eight programs in obesity and type 2 diabetes.

The deal, which was announced with AstraZeneca CEO Pascal Soriot accompanying U.K. Prime Minister Keir Starmer on a trip to China, includes an upfront payment of $1.2 billion for the rights to CSPC's experimental drugs outside of China. CSPC could receive another $3.5 billion based on development and regulatory milestones, as well as billions more if certain sales targets are hit.

The U.K. pharma's move is just the latest example of large drugmakers picking up experimental medicines from Chinese biotechs in recent years. In 2023, it licensed a weight loss drug candidate, which is now in Phase 2 trials, from another Chinese firm, Eccogene. AZ has also previously announced partnerships with CSPC on a cardiovascular disease drug candidate and on AI-enabled drug research broadly.

AZ, which has been building up its presence in China, its second largest market, announced further investments in the country on Thursday.

podcast

The high stakes for Huntington's patients

Biotech company UniQure is set to meet with the Food and Drug Administration to discuss the path forward for its gene therapy for Huntington's disease, and the outcome could be potentially devastating for patients.

That's what patient advocate Lauren Holder says on the latest episode of "The Readout LOUD," STAT's weekly biotech podcast. Holder joins the podcast for an in-depth discussion of how the regulator's changing perspective on UniQure's trial affects patients, and patients' attempts to petition the agency to reverse course.

Adam, Elaine, and Allison also chat about the latest news in the life sciences, including the new slate of drug prices negotiated by Medicare, a rise in pharmaceutical industry lobbying, and a halt on another gene therapy trial.

Listen here.

drug pricing

Trump officials pitch drug price plan to senators

Trump administration health officials met privately with Republican senators this week to press for legislation that codifies the president's "most-favored nation" drug pricing idea, STAT's Daniel Payne writes. This would tie U.S. Medicaid prices to the lowest paid in other wealthy countries.

CMS Administrator Mehmet Oz and other officials offered few new details, emphasizing instead the core premise: Force drugmakers to lower U.S. prices and recoup losses overseas.

Some Republicans expressed skepticism, viewing the proposal as government price controls, though the meeting's tone was neutral. The push revives an idea Congress previously rejected, but it comes amid heightened attention as Trump personally urges lawmakers to act and some Republicans show increased openness to using federal leverage to curb drug costs.

vaccines

Sanofi shelves mRNA flu shots, shifts strategy

Sanofi has deprioritized its mRNA-based seasonal flu vaccine and does not expect to bring one to market anytime soon, FierceBiotech writes. It is scrapping a Phase 1 candidate as part of a broader strategic shift, rather than a one-off cut to its pipeline.

The company says it remains committed to mRNA technology, pointing to ongoing work on an mRNA pandemic flu vaccine with encouraging early data, but is refocusing its commercial flu efforts on established flu products.

Although Sanofi CEO Paul Hudson noted on a call with reporters the company doesn't "make clinical decisions based on politics or sentiment in the now," he noted how the broader change U.S. vaccine policy has already sparked change.

"You've seen some of the complexity around the change in leadership in health care in the United States," Hudson said. "We're not complaining, we just recognize that the vaccine coverage rates will have dipped a little because of the debates that are happening in the public."

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