FDA
The FDA granted the approval of Revolution Medicines’ daraxonrasib, now to be known as Rasonque, in pancreatic adenocarcinoma 6.5 months ahead of deadline.
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Real progress happens where unmet medical need is greatest and patient impact can be clearly measured. Pediatric rare cancers exemplify these criteria and have therefore generated continued investment, even in a capital-constrained environment.
If confirmed by the Senate, Heidi Overton—officially revealed Wednesday as President Donald Trump’s choice for FDA commissioner—is expected to be a boon for developers of psychedelic therapeutics and non-opioid pain drugs, but could tow the party line on vaccines.
Capricor Therapeutics’ CEO Linda Marbán believes the FDA is willing to work with the biotech as the Aug. 22 deadline approaches for its Duchenne muscular dystrophy cell therapy deramiocel, with the biotech’s stock climbing 68% on the update.
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Three fatalities occurring recently in Chinese clinical trials have spurred two members of U.S. Congress to urge the FDA to enact new policies designed to protect American patients.
Regeneron’s Pasatru is the second fibrodysplasia ossificans progressiva drug approved in the U.S., following the 2023 approval of Ipsen’s Sohonos.
The accelerated greenlight for Ultragenyx’s gene therapy for glycogen storage disease has raised analyst expectations for approval of UX111, which the FDA rejected last summer and is currently reviewing for a second time. A decision is expected by mid-September.
If Heidi Overton clears the Senate nomination process, analysts say she will have a tough job ahead to “restore predictability” to an agency battered by layoffs and controversial policies.
Amylyx shares good news for its investigational GLP-1 blocker; Definium announces positive data for its LSD-based therapy in anxiety; Summit/Akeso’s ivonescimab shows degrading survival benefits; patients treated with Neurocrine’s Prader-Willi drug experience serious adverse events; and Eli Lilly cracks down on the black market for next-gen obesity asset retatrutide.
The FDA approves Zenbexus just as copycat competitors depress sales of Bristol Myers Squibb’s multiple myeloma drugs Pomalyst and Revlimid, which crashed 71% and 49% year-on-year, respectively, in the second quarter.
Companies that manufacture MDMA and psilocybin for patients in other countries are looking to use their experience to access the potential U.S. market.
Taiho Pharmaceutical and Cullinan Therapeutics’ cancer asset is already under FDA review in a later setting, with a decision expected by February.
After two rejections, Replimune has made it across the regulatory finish line with its advanced melanoma therapy. Meanwhile, the last of the major pharmas, including Eli Lilly and Novo Nordisk, reported second quarter earnings, and psychedelics made headlines once again.
ITM Isotope Technologies plans on resubmitting its advanced cancer radiopharma therapy to the FDA after addressing the agency’s concerns.