UniQure plans to seek approval for Huntington's therapy still blocked by FDA

Another FDA meeting, another setback for UniQure and its promising gene therapy for Huntington's disease. The treatment, called AMT-130, once appeared on a speedy path to approval, but is now in regulatory limbo.

Following a meeting at the end of January, FDA officials told UniQure that data from a completed single-arm clinical trial of AMT-130 were not sufficient to support the filing of a marketing application. This is the same conclusion reached by the FDA after a meeting last November.

The FDA wants UniQure to conduct a new clinical trial that compares AMT-130 to a sham surgery, the company said.

UniQure is among a growing number of companies developing treatments for rare diseases that have encountered regulatory setbacks or outright rejections from the FDA, suggesting the agency is setting a higher bar for approval of the medicines.

Drug pricing

How Trump's 'most-favored nation' gambit is impacting Europe

President Trump's push to force wealthy European countries to pay more for medicines — so, in theory, the U.S. can pay less — has injected fresh uncertainty into Europe's already fraught drug-pricing ecosystem. While prices haven't suddenly spiked, the broader industry view is that if Washington moves toward most-favored nation pricing, costs will rise abroad, STAT's Andrew Joseph writes from London.

"You can imagine over time lower prices in the U.S. but higher prices in a number of countries," AstraZeneca CEO Pascal Soriot told reporters at an event last month.

Some companies are now hinting they may avoid certain markets altogether if it's no longer being cost-effective to be there, particularly as European health authorities begin scrutinizing their current drug spending more carefully.

public health

Vaccine 'godfather' Stanley Plotkin is aghast

Stanley Plotkin, who is now 93, spent decades watching children die of diseases we now barely remember — and then helping to build the vaccines that made those deaths rare enough to become news. Plotkin, once the head of vaccine development at Pasteur-Mérieux (now Sanofi Pasteur), helped develop the rubella vaccine and rotavirus vaccine, among others. His textbook is the field's bible.

But speaking with STAT's Helen Branswell, he bemoaned the situation he sees today: declining vaccination rates, anti-vaccine leadership inside HHS, social media misinformation, and a generation that has never seen measles wards or rubella-stricken newborns. His view is unsparing:

"To tell people that they don't need to be vaccinated is simply promoting disease," he said. "Not only is it stupid, but it's immoral."

Cancer

A Merck drug to watch

From STAT's Matthew Herper: Merck presented two positive studies of its drug Welireg, generically known as belzutifan, to existing regimens in renal cell carcinoma at the American Society of Clinical Oncology Genitourinary Cancers Symposium. In one, adding Welireg to Lenvima, another Merck drug, reduced disease recurrence or death by 30% in patients who had progressed after immunotherapy compared to Cabometyx, a drug made by Exelixis. In a second, combining Welireg with Keytruda reduced disease recurrence or death by 28%.

Daina Graybosh, an analyst at Leerink Partners, wrote in a note to clients that the data will be a new standard of care in adjuvant and second-line kidney cancer: "We believe the strength of outcomes and revenue outlook for belzutifan will surprise most Merck investors and analysts, who have been overlooking belz qualitatively and in their revenue projections." Graybosh also wrote that the data could be a positive for Arcus Biosciences, which is developing a similar drug that is believed to be more potent.

drug pricing

Some White House drug-pricing deals have time limits

SEC filings reveal that at least some of President Trump's "most-favored nation" drug-pricing deals last just three years, terms that were previously undisclosed.

STAT's John Wilkerson writes reviewed SEC documents for the 16 companies that reached agreements with President Trump, and two reported that they would expire after three years. Merck's 2025 filing states that it "entered into a three-year agreement (the MFN Agreement) with the U.S. government." Sanofi also disclosed that its "MFN agreement is only for a duration of three years."

Amgen didn't give an exact timeline for its deal, but it cautioned investors that the administration seeks to codify the arrangements into law, which could apply to more products, payers, or pricing arrangements "for a longer period than those resulting from the MFN Terms."

legal

Novartis settles Henrietta Lacks lawsuit

Novartis has quietly settled a lawsuit brought by the estate of Henrietta Lacks, the Black woman whose cervical cancer cells were taken without her knowledge in 1951. Her cells became the first immortalized human cell line, underpinning decades of biomedical advances from the polio vaccine to genetic mapping and Covid-19 research.

Terms of the agreement, finalized last month in federal court in Maryland, were not disclosed. Both the Lacks family and the Swiss drugmaker declined further comment beyond confirming the resolution.

The case is the second such settlement after a 2023 deal with Thermo Fisher Scientific, as the estate continues to pursue other pharmaceutical companies, including Ultragenyx and Viatris, alleging they unjustly profited from "HeLa" cells cultivated from what the complaint described as "stolen" tissue.

opinion

Democratizing negligence with at-home ketamine?

Telehealth ketamine companies have reframed mail-order Schedule III narcotics as "access." But in reality, the practice often amounts to outsourcing critical medical oversight to vulnerable patients, who can self-assess, self-administer, and self-dose potent medicines, opines health author and independent researcher Michael Alvear.

Drawing on a six-month analysis of Reddit forums and his own remission experience with clinic-based Spravato, he describes widespread dosing confusion, unmanaged side effects, and patients crowdsourcing safety advice in the absence of real-time monitoring.

Although Alvear notes that online complaints are not prevalence data, he argues they function as early warning signals in a decentralized system largely outside mandatory adverse-event reporting. With tens of thousands of patients receiving at-home ketamine under extended federal telehealth flexibilities, he warns that stripping away supervision — standard in every other ketamine context — shifts the burden of risk management onto patients themselves. That, he concludes, is not democratized care. It's democratized negligence.

More around STAT