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Morning. Today, we discuss how Duchenne patients and their families are responding to the shelving of Sarepta Therapeutics’ gene therapy, see an FDA panel made up largely of SSRI skeptics lambast their use in pregnancy, and more.
The need-to-know this morning
European regulators said Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy should not be approved, further imperiling the future of the treatment, which is also facing increasing scrutiny in the U.S. Roche has commercial rights to the gene therapy, called Elevidys, in Europe and other countries.
European regulators said Eli Lilly’s Alzheimer’s treatment Kisunla should be approved for a select group of patients, changing course from an initial recommendation to reject the drug.
Bristol Myers Squibb named Cristian Massacesi as its new chief medical officer. Massacesi is a phyisican and most recently served as chief medical officer at AstraZeneca and its Alexion unit. He replaces Samit Hirawat, who served as Bristol’s CMO for the past six years.
Families devastated after FDA halts Sarepta gene therapy
Families of boys with Duchenne muscular dystrophy were devastated when the Food and Drug Administration asked Sarepta Therapeutics to halt shipments of its gene therapy, Elevidys, citing safety concerns — and the company subsequently agreed earlier this week. Parents who had fought for months to secure access are now stuck in limbo, with appointments abruptly canceled and little communication from regulators, STAT’s Jason Mast reports.
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