More drama involving UniQure, its Huntington's treatment, and the FDA

A senior FDA official spoke (anonymously) late yesterday to my colleague Adam Feuerstein, explaining in more detail why the agency decided to block UniQure from submitting a marketing application for its Huntington's disease treatment — a story we brought to you Monday.

Agency reviewers "are not convinced there's any therapeutic benefit of the product," the official said. "If we felt there was any therapeutic benefit, we, of course, would approve it. But they're not persuaded," the official added.

UniQure CEO Matt Kapusta also talked to Adam last night. Kapusta said the company was "incredulous that a senior FDA official is speaking to the media and communicating things that in many respects, have never been communicated to us."

"We've had five meetings with the FDA in the last 15 months, and a number of the things that were stated [by the FDA official] are borderline confidential information, and were never communicated to UniQure in any of the written feedback or otherwise. It's highly concerning to us," Kapusta added.

There's a lot more detail in Adam's insider account of this fight between UniQure and the FDA. Unfortunately, people with Huntington's disease are caught in the middle of it all.

health tech

What a new chatbot tells us about FDA's regulation of AI tools

FDA Commissioner Marty Makary has stressed his desire to bring artificial intelligence tools to patients, but it hasn't been clear how the agency will regulate rapidly evolving uses of the technology.

My colleague Katie Palmer dug into one new example that could offer some clues. In November, the FDA quietly handed a breakthrough device designation to a chatbot for patients recovering from joint replacement surgery. It's developed by a company called RecovryAI, which is coming out of stealth today as it announces the designation.

The chatbot would be prescribed to patients to use in the 30 days after surgery, and it will encourage them to check in twice a day about their sleep, activity, diet, and other elements of recovery.

politics

CMS wants more drugmakers to join Medicaid 'most-favored nations' pilot

It seems pharma companies may not be scrambling to join a Trump administration pilot program aimed at bringing "most-favored nations" pricing to Medicaid.

CMS said yesterday it's extending the deadline for drugmakers to apply to the Generating Cost Reductions for U.S. Medicaid Model, or the "GENEROUS" program, from the end of this month to the end of next month. The idea is companies would offer drugs to Medicaid at prices in line with what other countries pay. Participation is voluntary for both drugmakers and states.

This approach to Medicaid pricing was part of the deals that major pharma companies signed with the Trump administration last year that allowed them to be exempt from tariffs.

Though the administration has touted the policy as part of its efforts to lower drug costs, experts have been skeptical this policy would save states much money. Medicaid programs already get the lowest drug prices in the U.S. compared to all other payers. The impact on out-of-pocket costs for Medicaid enrollees would also likely be limited, since they already pay very little or nothing for drugs.

gene editing

FDA lifts clinical hold on Intellia's heart therapy

Intellia Therapeutics said yesterday it will resume Phase 3 trials of its CRISPR gene-editing therapy after the FDA removed a clinical hold.

The company has been studying its therapy, called nex-z, in two forms of a disease called transthyretin amyloidosis (ATTR), one that affects the heart and one that affects the nerves. It paused the two trials after a patient in the heart study was hospitalized with liver damage.

The FDA in January lifted the hold on the nerve study and is now also doing so with the heart study. Intellia has agreed that going forward, it will conduct enhanced monitoring of liver laboratory tests and exclude patients with certain liver abnormalities. In the heart study, it will also exclude patients with a recent history of cardiovascular instability and those with an ejection fraction less than 25% at the time of screening.

Liver damage has been a known risk for many CRISPR-based medicines in development, in part because they often involve trying to knock out or modify a gene in that organ.

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