Hello, everyone, and welcome to the middle of the week. Congratulations on making it this far, because it is an accomplishment, after all. The next step, of course, is to somehow forge ahead. And why not? Just consider the alternatives. On that optimistic note, please join us for a needed cup or three of stimulation. Our choice today is salted caramel, which reminds us of the breezy Jersey shore. Meanwhile, here are some items of interest to get you going. Have a wonderful day, and do drop us a line when you hear something juicy. …
A young boy died in a trial for an experimental Pfizer gene therapy for Duchenne muscular dystrophy, STAT writes, citing a note that was posted online by Parent Project Muscular Dystrophy and confirmed by the company. The boy, who was enrolled in a trial studying the treatment in boys aged 2 or 3, had received the therapy early last year and died of cardiac arrest. The company said it was still working to understand what happened and the exact cause. Deaths and severe side effects conclusively linked to gene therapy have generally happened soon after dosing. But cardiac effects have been seen before in gene therapy studies.
A U.S. Food and Drug Administration advisory panel will meet on June 10 to review an application by Eli Lilly to market its experimental Alzheimer’s disease drug, donanemab, Reuters says. The drug faced two separate regulatory delays in the U.S., while a similar therapy by Eisai and partner Biogen, called Leqembi, received FDA approval last year. In January, the FDA declined to assign accelerated approval to the Lilly drug and further delayed approval in March by scheduling the advisory meeting. Experts have said an association with side effects such as brain swelling and bleeding could pose a challenge to approval. Three people who were on the treatment in the company’s trial died.
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