FDA
A late-stage test of Roche and Ionis Pharmaceuticals’ sefaxersen showed that the drug reduced protein in the urine, which suggests the RNA-targeting therapy could improve kidney function and delay transplant.
Novo plans to hit $23B in revenue by 2035, has further reduced its workforce by 4,000 and is boosting manufacturing capacity for its Wegovy pill, but investors are unconvinced it will be enough to turn things around; Viking Therapeutics and Roche report positive results for their weight loss assets; Ultragenyx snags FDA approval for its Sanfilippo syndrome gene therapy; IPOs keep on coming.
As Chinese companies push beyond “me-too” drugs and the country lays out a new five-year plan targeting a $522 billion–strong biotech industry by 2030, experts say more predictable funding and regulation could help the U.S. maintain its edge.
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.
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?
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.
BioSpace takes stock of the psychedelic therapeutics space, which is once again on the cusp of its first FDA approval.
The FDA held a public forum on Monday to discuss the future of psychedelic therapeutics, followed by a meeting on Wednesday regarding recommendations for reauthorizing the Prescription Drug User Fee Act the day after launching its TrialBlazer initiative. Meanwhile, Definium Therapeutics notched another late-stage trial win for its LSD drug, and Novartis continues to reel from the company’s triple asset stumble.
The FDA aims to leverage the expertise of qualified research institutions paired with drug sponsors to lessen the risk of clinical holds during the investigational new drug review process and expedite the path to first-in-human development in the U.S. The initial application period will close on Oct. 30.
The FDA gathers Wednesday to hear what industry stakeholders and the public want included in the latest iteration of the Prescription Drug User Fee Act that sets the agency’s priorities for fiscal years 2028–2032.
PRESS RELEASES