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
While Novartis and Bayer got there first, AstraZeneca, Bristol Myers Squibb and Eli Lilly are all vying to bring their radiopharmaceutical assets to a market projected to be worth over $13 billion by 2033.
In the coming two weeks, the FDA is expected to announce three big decisions, including one for a dry eye disease therapy.
The European Union’s CHMP said that the benefits of the drug, already approved in the U.S., do not outweigh the risk of potentially fatal brain swelling and bleeding.
Compounded versions could make up as much as 40% of the semaglutide market, said Novo Nordisk CEO Lars Fruergaard Jorgensen on Thursday, but the company hopes to win patients over.
Milestone Pharmaceuticals hit another bump in the road in its quest to get Cardamyst approved for paroxysmal supraventricular tachycardia when the FDA issued a Complete Response Letter on Friday.
Lexicon’s LX9851 targets ACSL5, a liver enzyme involved in fat metabolism that helps moderate fat accumulation and slow down gastric emptying.
Arbutus is also exiting its corporate headquarters in Pennsylvania and will terminate all in-house scientific research. The company’s focus is now an RNAi asset for hepatitis B.
BNT327, a PD L1/VEGF antibody, belongs to a class of next-generation immunotherapies hoping to beat out Keytruda.
Merck’s new formulation of the mega-blockbuster Keytruda, made in collaboration with Alteogen, could help to keep the drug’s patent cliff at bay.
Deloitte urged pharma executives to “be bold” in a new report tracking the top 20 pharmaceutical companies’ R&D performance.