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The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
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With the biopharma industry performing better of late, analysts, executives and other industry watchers are “cautiously optimistic”—a term heard all over the streets of San Francisco at the J.P. Morgan Healthcare Conference earlier this month.
Bristol Myers Squibb, GSK and Merck are contributing drug ingredients as part of their deals with the White House but are keeping many of the terms of their agreements private.
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.
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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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For an exclusive license to the preclinical Lp(a) disruptor, AstraZeneca is paying CSPC Pharmaceutical Group $100 million upfront and offering up to $1.92 billion in regulatory and commercial milestones.
J&J has recently pulled back from the infectious diseases space, including winding down R&D activity in this area in August 2023.
The clinical hold doesn’t cover its drug’s Investigational New Drug application for autoimmune hepatitis, for which the Phase IIa PORTOLA trial is ongoing.
After several high-profile failures, including BMS’ $1.5B breakup with Agenus, anti-TIGIT therapies are generating cautious optimism.
After psychological side effects doomed the first generation of cannabinoid receptor 1–targeting drugs for weight loss, Novo Nordisk, Corbus Pharmaceuticals and Skye Bioscience are betting that a new mechanism of action will improve the safety profile.
Relay Therapeutics is cutting its workforce to help streamline its research organization as it looks to complete its first large-scale, pivotal clinical trial.
Scaling GLP-1 manufacturing capacity remains a key priority for the pharma industry, to help supply catch up with the insatiable demand for weight loss drugs.
Under the deal announced Friday, the Italian pharma will make an upfront payment of $825 million to Sanofi for global rights to a biologic for the treatment of cold agglutinin disease, with milestone payments of up to $250 million.
The company is lowering its previously issued guidance for 2024 adjusted earnings per share by $0.04 per share to account for acquired in-process research and development expenses incurred for acquisitions and collaborations in the third quarter.
Opdivo’s approval for patients with resectable non-small cell lung cancer comes as the regulator recently raised concerns of overtreatment with this type of therapeutic regimen with platinum-doublet chemotherapy.