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STAT+: Roche to launch another Elevidys trial, with eyes on European approval
In an attempt to win European approval for the controversial medicine, Roche said Thursday it would run another trial of the Duchenne muscular dystrophy gene therapy Elevidys.
The Swiss company’s move comes after European regulators last year gave a negative review to the therapy, saying it had failed to demonstrate long-term benefits for patients with the degenerative muscle condition. Roche has rights to the therapy outside the U.S., where it is marketed by its developer, Sarepta Therapeutics.
Roche said the Phase 3 trial will generate the type of evidence that could lead to a resubmission with European officials and to applications with regulatory agencies in other parts of the world. The study will evaluate the safety and efficacy of Elevidys versus placebo over 72 weeks in roughly 100 boys at the early stages of the disease.
In an attempt to win European approval for the controversial medicine, Roche said Thursday it would run another trial of the Duchenne muscular dystrophy gene therapy Elevidys.
The Swiss company’s move comes after European regulators last year gave a negative review to the therapy, saying it had failed to demonstrate long-term benefits for patients with the degenerative muscle condition. Roche has rights to the therapy outside the U.S., where it is marketed by its developer, Sarepta Therapeutics.
Roche said the Phase 3 trial will generate the type of evidence that could lead to a resubmission with European officials and to applications with regulatory agencies in other parts of the world. The study will evaluate the safety and efficacy of Elevidys versus placebo over 72 weeks in roughly 100 boys at the early stages of the disease.
WASHINGTON — President Trump moved on Saturday to “reverse the crisis of serious mental illness in America” by boosting access to psychedelic drugs in clinical settings. In an executive order, he directed the federal government to rush access to treatments and reevaluate their status as controlled substances.
The order directs the Food and Drug Administration to expedite some psychedelics as breakthrough drugs, as well as allowing them to be used through right-to-try legislation, which allow terminally ill patients to try experimental drugs outside of usual regulatory pathways.
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Optimism for Trump’s CDC pick is tempered by questions about RFK Jr.’s role
The nomination of a new leader for the Centers for Disease Control and Prevention — one who has scientific credentials and no public ties to the anti-vaccine movement — has generated sighs of relief in the public health world.
As one CDC employee, who asked not to be named, put it on Friday, among staff “the general vibe is guarded but hopeful.”
The nomination of a new leader for the Centers for Disease Control and Prevention — one who has scientific credentials and no public ties to the anti-vaccine movement — has generated sighs of relief in the public health world.
As one CDC employee, who asked not to be named, put it on Friday, among staff “the general vibe is guarded but hopeful.”
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RevMed’s stunning success; FDA to reclassify peptides; and more
RevMed’s stunning success; FDA to reclassify peptides; and more
Welcome back to Endpoints Weekly! Q1 earnings season is officially upon us. Max Gelman kicked off our coverage this week with a story about Johnson & Johnson’s confidence in navigating biosimilar competition … Read More
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