FDA decides to hold ad comm for Sarepta after all

The FDA is changing course and now wants to hold an advisory committee hearing to deliberate over Sarepta's experimental gene therapy for Duchenne muscular dystrophy. Just weeks ago, the company said the FDA didn't deem a meeting of outside experts to be necessary — which surprised patients and analysts alike. The ad comm date hasn't been posted yet, but will be in advance of the treatment's May 29 PDUFA date.

Sarepta CEO Doug Ingram told investors that the agency didn't raise any safety concerns about the treatment, called SRP-9001 — and that the hearing will likely focus on efficacy. The drug is under review for an accelerated approval, and Ingram said the evidence is "very robust, far exceeding, we think, the standard."

But patient advocates are glad the FDA is taking this extra measure of caution. "For the better of the Duchenne community, it's important for everyone to understand the data that goes into the decision for a treatment that can be so transformative," one told STAT. "At this point, we have limited visibility into the data and the long term risks for this vulnerable population."

marketing

Novo Nordisk suspended by U.K. trade group for 'serious breaches'

A U.K. pharma trade group has suspended Novo Nordisk for two years for "serious breaches" that involve the marketing of its weight-loss drug, Saxenda. The final straw for the Association of the British Pharmaceutical Industry was when it received a complaint that Novo Nordisk had sponsored weight management courses on LinkedIn for health professionals, without explicitly saying that it would be a sponsor.

The self-regulatory arm of the trade group said it was "very concerned that Novo Nordisk did not recognize that this was a large-scale Saxenda promotional campaign, which Novo Nordisk knowingly paid for and which was disguised." Novo Nordisk ultimately violated seven of the trade group's codes of practice, which were deemed too "likely to bring discredit on, or reduce confidence in, the pharmaceutical industry."

readout loud

Is AI dictating patient care?

What happens when your bank falls apart? And can Illumina endure some shareholder activism? We cover all that and more this week on the 250th episode of "The Readout LOUD," STAT's biotech podcast.

STAT reporter Bob Herman joins us to explain how treatment algorithms powered by artificial intelligence are being used more frequently by Medicare Advantage plans to deny claims, even when continued treatment is medically justified. We'll also discuss the latest news in the life sciences, including the continued fallout of the run on Silicon Valley Bank, the return of Carl Icahn, and a long-awaited pharmaceutical megadeal.

Listen here.

Clinical research

Califf wants insurers to support post-market research

FDA Commissioner Robert Califf says that insurance companies need to step up and contribute to the post-approval clinical research on drugs. He added that clinicians report that it's "very hard to do research in the current environment." Califf, who has conducted scads of clinical studies himself, is on the lookout to modernize the way drugs are tested. He's particularly keen on integrating real-world evidence into the system of validating a drug's efficacy and safety.

"I looked at the profits, by the way, on the internet before coming over, and there's a lot of money being made in this business," Califf told AHIP President Matt Eyles in front of an audience of payers. "And it would seem like we ought to all be working together to develop the evidence so we spend the money on the things that work and we don't spend the money on the things that don't work."