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Following a tumultuous stretch under ex-Commissioner Marty Makary, current FDA leadership is working to restore reliability, integrity and transparency to the agency—but experts say that doesn’t necessarily mean an easier regulatory road for drugmakers.
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.
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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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The FDA has placed a clinical hold on RGX-121 after small nodules or cystic masses were detected in the spines of five patients enrolled in REGENXBIO’s registrational trial. The biotech no longer plans to resubmit the candidate—which was rejected in February—in the near term.
Roche, through its subsidiary Genentech, will gain access to the investigational asset HM17321, which is designed to not only elicit weight loss but also improve lean mass.
The current classifications of vaccine recommendations could imply that not all shots require the consent of patients or their parents, according to Health Secretary Robert F. Kennedy Jr.
Not much is known about Boehringer Ingelheim’s drug candidate, dubbed BI 3031185, though the asset has completed at least two other Phase 1 studies this year.
Kaos Capital in a letter to other Capricor Therapeutics shareholders called for “an immediate meeting, board change, and capital-preservation plan.” The biotech recently put all other pipeline work on pause as it awaits an Aug. 22 PDUFA date for embattled cell therapy deramiocel.
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.