FDA
After cardiac safety signals led to a full clinical hold on Aardvark Therapeutics’ studies of ARD-101, the biotech conducted an additional safety study, in which no cardiac events were detected. Aardvark plans to resume testing the candidate next year.
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Leveraging recently proposed regulatory flexibilities, the gene-editing sector is working to cut R&D costs and unlock indications that were previously economically unviable, according to a new playbook published by the Alliance for Regenerative Medicine.
The FDA has taken important steps through Operation TrialBlazer and stronger scrutiny of foreign clinical data, but more must be done to make the United States more competitive in attracting clinical trials as China’s capabilities rise.
The approval of Mirum Pharmaceuticals’ ALK2 inhibitor for fibrodysplasia ossificans progressiva on Friday adds a third option for patients who had long been without any treatments, joining Regeneron’s recently approved Pasatru and Ipsen’s Sohonos.
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Following several recent initiatives, the FDA’s newest rare disease framework sets out to standardize the agency’s approach to scientific evidence and bring rare disease therapies to patients faster.
After one patient death prompted an FDA hold this spring, Newron Pharmaceuticals has now shared that a total of four fatalities have been recorded among participants receiving its schizophrenia candidate. A fifth death was recorded among those taking the placebo.
Despite the regulatory delay, Novo is still expecting a 2027 launch for its hemophilia A drug denecimig.
Patients had adverse events after receiving a Fresenius Kabi medicine that experienced manufacturing deviations.
Amid rising industry concerns about U.S. competitiveness with China, the Trump administration is rolling out four ARPA-H initiatives to modernize clinical trial design, site activation, consent and patient data collection.
Ebvallo, which Pierre Fabre Pharmaceuticals and Atara Biotherapeutics are proposing to treat a posttransplant complication, joins a wave of rejected or discouraged therapies on which the FDA has reversed previous guidance after the exit of former Commissioner Marty Makary.
Summit Therapeutics scores a $2 billion equity investment from AstraZeneca to support ivonescimab’s late-stage development; uniQure crashes after four-year data from its Huntington’s disease gene therapy failed to impress; Kyverna Therapeutics brings new light to a struggling CAR T space in autoimmune disease.
In the Phase 3 TEMPO program, AbbVie’s Juvmo elicited significant improvements in motor symptoms and prolonged periods with good symptom control.
FDA inspectors found evidence that the compounding pharmacy was making versions of Eli Lilly’s tirzepatide and Novo’s semaglutide “regularly or in inordinate amounts” at a site with “insanitary conditions.”
In Thursday’s Senate committee meeting, FDA commissioner nominee Heidi Overton was repeatedly questioned about her ability to retain the FDA’s independence under President Donald Trump, as well as about her views on vaccines and abortion drugs.