News
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.
FEATURED STORIES
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.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
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.
THE LATEST
BioSpace spoke to talent acquisition leaders about how they think artificial intelligence will shape the future of their function.
Analysts expect the partners’ first-mover advantage, clinical data and existing presence in lung disease to translate into significant sales, with GlobalData predicting Dupixent’s COPD revenues will top $6.5 billion within 10 years.
Evonik’s latest layoffs are tied to discontinuing production of keto acids in Hanau, Germany, by the end of next year. Earlier this year, news broke the company is also cutting up to 2,000 employees globally by 2026.
Under the deal, the Danish pharma will gain access to Longboard’s 5-HT2C receptor superagonist that is currently in late-stage development for seizures in various developmental and epileptic encephalopathies, including Dravet syndrome.
European CDMO Ardena will buy Catalent’s oral solids manufacturing facility in Somerset, N.J.
While the regulator conducts another review into the supply of Eli Lilly’s tirzepatide, compounders will be able to continue selling their own remixed versions of the blockbuster drug.
The approval makes Pfizer’s Hympavzi the first once-weekly subcutaneous prophylactic injection for hemophilia B in the U.S., according to the company, which is currently embroiled in a row with activist investor Starboard Value.
While ex vivo genome editing results in highly effective cell therapies, it can lead to off-target effects. Caribou Biosciences has come up with a novel approach for potentially more precise gene editing compared to all-RNA guides.
The FDA is looking at four events for the remainder of October, one of which is an advisory committee meeting for a dual SGLT inhibitor for use alongside insulin in type 1 diabetes and chronic kidney disease.
Since its inception in 1992, the FDA’s accelerated approval pathway has helped shepherd nearly 300 new drugs to the market. However, recent years have seen a number of high-profile market withdrawals and failed confirmatory trials.