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Roche said the Phase II results help build the case for advancing CT-388 into late-stage testing, which is set to get underway this quarter.
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Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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Merck’s Winrevair is the second PAH drug to get the FDA’s green light in the past week, following Johnson & Johnson’s Opsynvi, which won approval on Friday.
Health economics and outcomes research (HEOR) is a critical but sometimes overlooked part of drug development. Decentralized trials now make it easy.
Brazil’s Ministry of Health and nonprofit Caring Cross announced a collaboration Tuesday aimed at local manufacturing of CAR-T cell and stem cell gene therapies at a much lower cost than Europe and the U.S.
Stoke Therapeutics reported results for Dravet syndrome studies showing clinically meaningful effects, including reductions in convulsive seizure frequency, supporting the potential for disease modification.
The early-stage study showed that Viking Therapeutics’ oral obesity candidate VK2735, a dual agonist of the GLP-1 and GIP receptors, elicited a 3.3% reduction in mean body weight. The company plans to start a Phase II trial.
AstraZeneca’s Alexion on Monday secured the fourth indication for Ultomiris, which can now be used to treat the rare autoimmune condition neuromyelitis optica spectrum disorder.
Axsome clinched a late-stage victory for its chronic sleep disorder candidate AXS-12, which demonstrated significant improvement in cataplexy—the sudden loss of muscle tone while a person is awake—after five weeks.
The National Institutes of Health claims BioNTech is in default regarding alleged royalty payments the agency contends it is owed in connection with the company’s COVID-19 vaccine Comirnaty.
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Regeneron’s bispecific antibody odronextamab was hit with Complete Response Letters from the FDA noting issues with the enrollment status of its confirmatory trials.