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After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
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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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The British pharma expects “meaningful” sales and earnings growth in 2024 and is upgrading its growth outlooks for 2026 and 2031, according to GSK CEO Emma Walmsley.
The U.S. Department of Health and Human Services on Thursday released new data showing that Americans pay three times more for prescription drugs than other developed countries and nearly 10 times more for insulin.
Alto Neuroscience and Fractyl Health provided further momentum to the recent spate of biotech initial public offerings, with both companies going public on Friday morning in respective $128 million and $110 million IPOs.
Kyverna Therapeutics, the fifth biotech with plans for an initial public offering this year, will use the proceeds to support the development of its anti-CD19 CAR-T therapies for autoimmune diseases.
The Centers for Medicare and Medicaid Services on Thursday sent its opening price proposals to drugmakers, which now have 30 days to either approve the offers or submit counteroffers.
Biotech companies are exploring multiple targets in the fight against neuroinflammation. Experts say these projects could yield the next breakthrough in treating Alzheimer’s disease.
The Swiss pharma on Thursday projected mid-single-digit sales growth for 2024, culling eight assets and looking to acquisitions and collaborations for de-risked assets with “significant potential.”
During a Wednesday webinar, FDA Commissioner Robert Califf said the agency is working on “systemic” changes to its advisory committee process.
Positive Phase III results for VX-548 could signal a new chapter in the fight against opioid addiction, but experts say there is still room to do better.
The first hearing on a potential injunction came the day before CMS is to release pricing proposals on 10 drugs.