matt’s take

On Vinay Prasad’s brief, chaotic tenure at the FDA

Vinay Prasad’s short and turbulent tenure at the FDA is ending the way much of it unfolded — in controversy.

In a new column, STAT’s Matthew Herper says that Prasad, whose upcoming departure from the agency was announced late Friday, was right that the FDA needed to demand stronger evidence before approving certain drugs, particularly in rare diseases. But his approach often caused chaos.

Prasad frequently escalated disputes that could have been resolved through normal regulatory channels, Matt says, and as STAT has reported, he overruled staff and alienated colleagues. His confrontational style, public fights with companies like Moderna, and controversial claims about vaccine safety also undermined his credibility.

Read more on Matt’s five lessons from the short-lived Prasad era.

FDA

Wall Street is also rendering a verdict on Prasad’s departure

From my colleague Adam Feuerstein: Gene therapy and rare disease drug stocks are trading higher in the early Monday session. UniQure, which may now have a path open for the filing of its Huntington’s disease treatment, rose 35%. RegenxBio, which saw its gene therapy for MPS II rejected by Prasad’s FDA center, was up 10%. Capricor Therapeutics rose 12%; Atara Biotherapeutics was up 22%.

“We see the departure of Dr. Vinay Prasad from CBER as the most significant news out of FDA this year, potentially driving an impactful shift in the agency’s regulatory culture from the last year of chaos. We see an improved regulatory outlook for names with complex therapies working in challenging, rare indications and expect a boost to a number of stocks in our coverage as a result,” said Baird biotech analyst Brian Skorney, in a note to investors Monday.

FDA Commissioner Marty Makary is in Miami this week speaking to health care investors attending a slew of broker conferences, noted Mizuho health care strategist Jared Holz. That should make for some interesting, perhaps awkward, conversations.

It took about 15 seconds for former FDA official Rick Pazdur’s name to rise to the top of the list of potential Prasad replacements. Whether that’s wishful thinking or a real possibility remains to be seen, but Pazdur is also in Miami. He spoke to an overflow room of investors on Sunday night at the Leerink Partners health care conference, sounding very much like a guy whose long, distinguished FDA career was interrupted, but not finished.

“Will [Prasad’s replacement] be someone with actual regulatory experience or a regulatory track record?” writes Stifel biotech analyst Paul Matteis, in a note this morning. “Our gut is that the Trump administration — with the midterms coming and the FDA on the radar of Congress — won’t want someone controversial, especially on vaccines. More to come, but one would think this all leads to FDA being more industry friendly.”

biotech

A strong Phase 3 trial for Xenon’s epilepsy drug

An experimental seizure drug called azetukalner made by Xenon Pharmaceuticals met the main goal of a Phase 3 trial in patients with focal onset seizures. It cut monthly seizure frequency by 53% compared with 10% for placebo over 12 weeks — results that seem to be stronger than results from the company’s earlier Phase 2 study, STAT’s Adam Feuerstein writes.

Xenon plans to file for approval with the FDA in the third quarter.

The therapy works through a Kv7 potassium channel mechanism not used by existing treatments. Beyond epilepsy, Xenon is also testing azetukalner in major depressive disorder and bipolar depression, which are far larger markets.

Read more.

from AXIOS:

A new response to health cyberattacks

By Peter Sullivan

Illustration: Aïda Amer/Axios

Two years after the seismic Change Healthcare cyberattack, Congress is advancing a plan to safeguard against the kind of hacks that can expose millions of people’s private data and cripple health systems. Why it matters: The bipartisan plan puts the burden on the government and providers to prevent the kind of breach that reverberates across the entire industry, jeopardizing patient access to needed treatments and costing hospitals billions. Driving the news: The Senate’s health committee late last month advanced legislation to fortify health care cybersecurity in a strong 22-1 vote. The bill would improve coordination among government agencies and requires HHS to develop an incident response plan. It also would establish new grants to health entities for cyberattack planning and response and make them use multi-factor authentication and encryption — a key shortcoming exposed by the Change breach. The average cost of a data breach in the U.S. is around $10 million, with health care having the highest breach cost among all industries, according to an IBM report. With hackers often stationed overseas, law enforcement has limited ability to respond. Between the lines: Democrats put forward legislation in 2024 in the wake of the Change attack that would have taken a more aggressive approach to cybersecurity requirements and penalties, prompting objections from the health industry. The legislation that advanced last week is seen as more of a middle ground that could have a better chance of advancing. Hospitals aren’t publicly commenting on specifics but could raise objections to some of the requirements in the bill. A spokesperson for the American Hospital Association declined to comment. The big picture: The bigger obstacle is a packed congressional agenda and little time before the midterm elections. Backers likely would have to attach the cybersecurity bill to a bigger legislative vehicle, like a year-end government funding deal. Keep reading