In tirzepatide controversy, some see FDA 'blind spot'

Tirzepatide, the Eli Lilly GLP-1 drug sold as Mounjaro for diabetes and Zepbound for obesity, has been at the center of a controversy that sparks questions about the FDA's process for tracking drug shortages.

After almost two years, the FDA declared an end to the shortage of tirzepatide earlier this month, which should have prohibited compound pharmacies from making copies of the drug. However, some traditional retail pharmacies said supplies of Lilly's treatment were still unavailable, and a trade group representing compounders took the FDA to court.

The problem, some experts say, is that the agency has an imperfect system for determining when shortages are resolved.

"Demand increases are a blind spot just because there's no real good marker of demand," a senior director at the American Society of Health-System Pharmacists told STAT. "The FDA has a very clear process for when it's a supply disruption causing a shortage, but when it's a demand increase, perhaps looking at some other available data will help inform when to take it off the list."

rsv

Hurdles in RSV vaccine age expansion

From STAT's Helen Branswell: Two of the three makers of RSV vaccines for adults — GSK and Pfizer — have been pushing hard to try to increase the market for their shots by targeting younger adults. In June the FDA expanded GSK's license to include adults ages 50 to 59 at high risk of severe disease. This week the agency granted Pfizer's application to expand the age range for its vaccine to include immunocompromised adults ages 18 to 59. But the companies have run up against a barrier: the Advisory Committee on Immunization Practices.

ACIP, which advises the CDC on vaccination policy, declined to recommend either vaccine for younger adults at its meeting this week, citing concerns the two vaccines may be linked to a risk of developing Guillain-Barré syndrome. (There's no signal of a GBS link with Moderna's RSV vaccine.) It's not that the group voted to reject an age extension; it didn't bring the issue up for a vote. Members of the ACIP subcommittee for RSV vaccines said that while the benefits of the shots outweigh the GBS risk for older adults, they want more evidence before they will consider recommending use in younger adults. Insurance companies are unlikely to pay for these vaccines for adults under the age of 60 without an ACIP recommendation.

Pharma

Sanofi third-quarter earnings top estimates

From STAT's Andrew Joseph: A year ago, Sanofi announced it was upping its spending on its research programs and, at the same time, was withdrawing future earnings guidance. The market reaction was brutal, with shares dropping by almost a fifth that day.

But since then, the company's share price has recovered. And today, the French pharma reported third-quarter earnings that beat analyst expectations, with operating income up 14.4%. Some of that was due to what the company called "earlier-than-anticipated" deliveries of its flu vaccine, but Sanofi also reported a 23.8% increase in sales of its inflammation-targeting drug Dupixent and strong sales of Beyfortus, which is used to protect babies from RSV and is rolling out in more countries. Earlier this week, Sanofi also raised its annual profit forecast.

"We are clearly moving forward" with the plan announced last October, CFO François Roger said on a call with reporters this morning. As an example of what Sanofi's investments in R&D could lead to, he pointed to the recent regulatory approvals of Dupixent in COPD, which are expected to lead to even bigger sales of the blockbuster medicine that it markets with Regeneron.

podcast

About Pfizer's activist troubles

Will an activist investor campaign against Pfizer lead to big changes at the struggling pharma giant? Why are some experts questioning the regulatory standards used to approve gene therapies for Duchenne muscular dystrophy? What's the difference between Novo Holdings and Novo Nordisk, and will it impact a $16 billion acquisition of Catalent? And what lies ahead for Robert F. Kennedy Jr. and the Make America Healthy Again movement?

We talk about all that and more on this week's episode of "The Readout LOUD," STAT's biotech podcast. In our "Chatty Cathy" segment, we mull over Starboard Value's criticisms of Pfizer and what possible changes, including the replacement of CEO Albert Bourla, might lie ahead. We also discuss the impact that Pfizer's failed gene therapy in Duchenne may have on other companies developing next-generation treatments, and noodle over Novo Holdings' controversial plan to acquire Catalent, one of the world's largest contract drug manufacturers.

Listen here.

rare disease

Monopar CEO brings back Wilson disease drug

Monopar Therapeutics — a small biotech in the radiopharmaceuticals space — is dusting off a discarded rare disease drug from AstraZeneca. It has licensed ALXN-1840, a candidate meant to treat Wilson disease that Alexion, now an AstraZeneca subsidiary, paid $855 million to acquire six years ago. Monopar CEO Chandler Robinson helped discover the drug two decades ago when he was an undergraduate at Northwestern University, FierceBiotech points out.

In 2021, ALXN-1840 outperformed standard-of-care treatments for Wilson's disease in a Phase 3 study — and it looked like it would win approval. But two small Phase 2 studies were underwhelming, and AstraZeneca got cold feet and stopped development in its tracks.

"Due in no small measure to the testimonials Chandler received from clinical trial patients who reported benefit while on the drug for years, we decided that this was an opportunity Monopar needed to pursue," Monopar cofounder Chris Starr told Fierce.

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