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Wednesday, May 8 2024

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Young Patient In Duchenne Gene Therapy Study Died, Says Pfizer

Read recent pharmaceutical developments in 麻豆女优 Health News' Prescription Drug Watch roundup.

A young patient died due to cardiac arrest after receiving Pfizer's experimental gene therapy being tested in a mid-stage trial for a muscle-wasting disorder called Duchenne muscular dystrophy (DMD), the drugmaker told Reuters on Tuesday. "A fatal serious adverse event was reported as cardiac arrest for a participant in the Phase 2 DAYLIGHT study," a company spokesperson told Reuters in an emailed response. (Jain, 5/7)

Eli Lilly said on Tuesday a panel of independent U.S. FDA advisers will discuss its experimental Alzheimer's disease drug, donanemab, on June 10. Donanemab has faced two separate regulatory delays in the United States, while a similar therapy by Eisai and partner Biogen, called Leqembi, received the U.S. Food and Drug Administration's approval last year. (5/7)

Xofluza (baloxavir marboxil, BMX) appears to be more effective than oseltamivir (OTV, Tamiflu) in lowering the secondary attack rate (SAR) of flu, according to a new study based on transmission dynamics in Japanese households. The study was published yesterday in Influenza and Other Respiratory Viruses. (Soucheray, 5/7)

Novo Nordisk A/S is asking for more time to answer questions from US Senator Bernie Sanders on the prices of Ozempic and Wegovy, the company鈥檚 blockbuster drugs for diabetes and weight loss. 鈥淲e plan to respond to the latest inquiry in a timely manner; however given the tight turnaround requested, we will need additional time to develop our response,鈥 a spokesperson for the Danish drugmaker said Tuesday in an emailed response to questions. (Kresge and Muller, 5/7)

Drug development is essentially a long, expensive bet: 90% of drugs fail during clinical trials, goes one of the life science industry鈥檚 most oft-quoted statistics. But new ways of identifying and testing therapies, a more precise understanding of disease, and a renewed sense of urgency to address longstanding public health issues could change that dynamic, life science leaders argued this week. (Wosen, 5/7)

This is part of the Morning Briefing, a summary of health policy coverage from major news organizations. Sign up for an email subscription.
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