FDA
Inspection access is not “an administrative footnote,” leaders of key FDA divisions wrote in an editorial on Wednesday vowing to expand oversight of foreign clinical trial sites.
FEATURED STORIES
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.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
Gastroesophageal adenocarcinoma presents the biggest market opportunity for Jazz Pharmaceuticals’ Ziihera, according to Truist Securities, which forecasts peak sales of the drug to reach $2.5 billion.
Though the potential change is unconfirmed, media outlets reported Wednesday that one deputy commissioner would focus on the intersection between health and AI, while another will be dedicated to drugs.
FDA extends its review of Capricor Therapeutics’ Duchenne muscular dystrophy cell therapy as an unhappy shareholder advocates for change; Merck and Moderna tout positive data for their mRNA-based cancer vaccine; Trump nominates White House aide Heidi Overton as FDA commissioner; and the oral obesity market heats up with rivals Eli Lilly and Novo Nordisk going head-to-head.
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.
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.
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.