February 25, 2025

The need-to-know this morning

Alnylam Pharmaceuticals is presenting updates from its R&D pipeline at a meeting with investors and analysts, including details on next-generation RNA-silencing drug to target ATTR amyloidosis and neuroscience and cardiovascular diseases.

glp-1s

Lilly expands its Zepbound vial offering

Eli Lilly announced this morning that it will offer more doses of its obesity drug Zepbound in vials and lower the prices of the doses it already sells.

Lilly initially launched Zepbound, which was originally sold in injectable pens, in vials last year. The move was seen as a way for the pharma giant to combat the rise in compounding.

CEO Dave Ricks told STAT in January that Lilly was considering expanding its vial offering. He acknowledged that he thinks there are some patients who have turned to compounding due to "real cost pressures" with the branded treatments.

glp-1s

Hims says it will pull back on semaglutide — sort of

From STAT's Katie Palmer: In its fourth quarter earnings call yesterday, direct-to-consumer telehealth company Hims & Hers said that it would stop selling compounded semaglutide in the doses offered by Novo Nordisk, after the FDA on Friday said the shortage had come to an end.

But the company hasn't given up on compounding the drugs: It will continue to prescribe other forms of semaglutide "when there is clinical necessity for that personalization," said CEO Andrew Dudum — in Hims' view, mostly for those who have documented side effects like nausea on commercial doses of the drug.

Meanwhile, the saga over what can be compounded at all continues: The same compounding trade group that sued the FDA for its handling of the shortage of Lilly's GLP-1 drug filed a similar suit yesterday over the agency's semaglutide shortage determination.

pharma

Pfizer's controversial hire: a former FDA official

Pfizer announced yesterday that it has tapped Patrizia Cavazzoni, the former head of the FDA's drug center, as chief medical officer, my colleague Matt Herper reports.

The announcement immediately prompted criticism on social media about the "revolving door" between the FDA and industry. It's been common for high-level regulators to join the agency after working with or within regulated companies, and vice-versa, but this trend has come under particular scrutiny recently by newly confirmed HHS Secretary RFK Jr. and his supporters. Read more.

My colleague Adam Feuerstein had a lot of thoughts about this move. "It's only February, but my pick for the Worst Biopharma CEO of 2025 has already been decided: Albert Bourla of Pfizer," he wrote in a new column.

Pfizer, as well as the rest of the pharma industry, have already been bashed by RFK Jr. and his supporters for being too cozy with the FDA. And Bourla may have just given them more ammunition, Adam writes. Read more.

gene therapy

Regeneron reports promising update on its deafness therapy

From STAT's O. Rose Broderick: In an ongoing trial of Regeneron's gene therapy for a rare variant of congenital deafness, 10 out of the 11 kids who received a post-treatment assessment saw "notable improvements," according to data presented yesterday at an otolaryngology conference.

The ongoing Phase 1/2 trial is one of a handful of studies evaluating the safety and efficacy of gene therapy in children and adolescents with otoferlin variants, which affect 1% to 8% of deaf people globally.

Three participants experienced improvements that boosted their hearing levels to "nearly normal" or "normal," Regeneron said. There were no adverse effects reported that were considered to be related to the gene therapy, though one participant had not experienced any changes to their baseline hearing after 24 weeks post-dosing.

While the promising results only apply to a small, rare variant of congenital deafness, the research could have a profound impact: More than 12,000 kids are born with hearing loss in the U.S. every year. Deaf communities are skeptical of this research, but it may not matter. The therapy's success across multiple teams has researchers and investors hungry for more, and the ear often escapes the notice of the immune system, making it an ideal environment for gene therapy.

infectious disease

U.S. agencies still joined WHO's flu vaccine meeting

The CDC and the FDA have joined a meeting led by the WHO this week on to select the flu viruses for next winter's flu vaccines, despite the Trump administration's plans to withdraw from the global health agency, sources told my colleague Helen Branswell.

The administration put a ban on all interactions with the WHO, which means the CDC and FDA would have had to receive an exemption.

This type of WHO meeting is important since participants not only discuss seasonal flu vaccines, but also review what is happening with other viruses like H5N1 bird flu that pose a pandemic risk.