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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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Despite recent concerns about suicidality and other neuropsychiatric issues, a recent study has found that Novo Nordisk’s Ozempic (semaglutide) is associated with lower risks of dementia, cognitive deficit and nicotine misuse.
After a busy first half of 2024, several companies are expecting key data readouts in the neuropsychiatric and neurodegenerative disease spaces during the next six months.
Why I advocated on Capitol Hill this month for the renewal of the FDA’s Priority Review Voucher program
Halia Therapeutics, NodThera and Gain Therapeutics target neuroinflammatory processes in hopes of modifying the course of Parkinson’s progression.
The Federal Trade Commission criticized the business practices of pharmacy benefit managers this week, but drugmakers are also at fault for the high costs of medicines.
Akebia Therapeutics on Thursday said it regained full U.S. rights to its chronic kidney disease anemia drug Vafseo, which the biotech has priced at around $15,500 per year.
Immutep shares jumped nearly 20% on Friday after data showed its LAG-3 therapy—plus Keytruda—elicited strong response rates in head and neck squamous cell carcinoma patients in the front-line setting.
CVS Caremark’s recent decision to take AbbVie’s Humira off its major formularies has caused the blockbuster to lose more market share to biosimilars, according to the latest report from Samsung Bioepis.
The Senate on Thursday unanimously passed a bill aimed at limiting the number of patents drugmakers can introduce and making it easier for generic and biosimilar competitors to enter the market.
Pfizer said Thursday it is pushing ahead with a once-daily, modified-release formulation of its oral GLP-1 obesity therapy danuglipron, with dose optimization studies in the second half of 2024.