Regulatory
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.
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.
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.
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.
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.
Sponsors of the pivotal study behind Amgen’s rare disease drug Tavneos saw unblinded findings for the drug and re-adjudicated data from nine patients to make the drug seem effective, according to European regulators.
Reports of seven deaths in patients who had taken Neurocrine Biosciences’ Vykat and the official termination of Aardvark Therapeutics’ Phase 3 trial after cardiac complications marked a tough day for the Prader-Willi syndrome community.
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