FDA
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.
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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.
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.
Newly permanent CBER and CDER directors Karim Mikhail and Michael Davis are widely expected to continue the FDA’s efforts toward an adaptive framework for rare disease therapies. If approved by the Senate, will commissioner nominee Heidi Overton stay the course?
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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.
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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.”
The weekly insulin Onswik will now be available to U.S. patients after initial approvals in Europe, Mexico and Japan.
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Citing the urgent unmet need for patients with advanced biliary tract cancer, Compass Therapeutics will continue to seek an approval path for its drug after the FDA recommended the biotech conduct a new clinical study.
The market approval for Fayuvi, which was rejected in 2025 due to manufacturing problems, comes shortly after Ultragenyx got the FDA nod for its first gene therapy last month.
After reviewing Bausch & Lomb’s response to its observations, the FDA questioned whether the company is committed to implementing significant design remediations.
BioSpace takes stock of the psychedelic therapeutics space, which is once again on the cusp of its first FDA approval.