News
The FDA has granted a three-month extension for Capricor Therapeutics’ Duchenne muscular dystrophy hopeful deramiocel in order to review additional data and consider a refined indication. The new target action date for the therapy is November 22.
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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.
The FDA’s August approval of Takeda’s Orzeyful for treating the full set of narcolepsy symptoms initiated a new era in the sleep market.
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The FDA has been trying to mend fences with rare disease drug developers and patient groups since the departure of former Commissioner Marty Makary three months ago. But advisory committee meetings last week for Replimune and Capricor Therapeutics only created more confusion and frustration.
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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.
The merger, which would create a new company bearing Ambros’ name, will help support development of Ambros’ non-opioid painkiller for patients with debilitating limb injuries.
The Phase 3 study is evaluating two doses of Novo’s Wegovy pill in people with obesity or overweight in combination with a reduced-calorie diet and increased physical activity.
After committing $2 billion to a new North Carolina facility to boost capacity for its next-gen obesity candidates, Roche’s Genentech is putting down more cash to expand a device fill-finish site in Oregon.
Scribe Therapeutics’ one-time epigenetic treatment is designed to slash the adherence burden of chronic cholesterol-lowering therapies while sidestepping the irreversibility of gene editing.
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.
Investors had been pessimistic about the probability of success for the personalized cancer vaccine intismeran autogene, so the Phase 3 win was a welcome surprise.
In this episode of Denatured, you’ll hear from Ian McGowan, Chief Medical Officer at Synklino and James Hunter, associate professor of transplantation at Oxford University. We explore transplantation and antiviral innovation and why CMV remains one of the most persistent and consequential challenges facing transplant patients today.
Melanoma was slower to recur in patients receiving Merck and Moderna’s mRNA vaccine with Keytruda, supporting use of the combination in adjuvant treatment. The partners plan to take the data to the FDA.