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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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Even as Biogen and Eisai’s Leqembi and Eli Lilly’s Kisunla slowly roll out onto the market, experts question the efficacy of these anti-amyloid antibodies and the amyloid hypothesis overall.
Blackstone and Bain Capital are said to be among the final bidders for the Japanese company’s Mitsubishi Tanabe Pharma, sources told Reuters Friday.
Following news of RSV lower respiratory tract infections in infants immunized with Moderna’s investigational RNA vaccines, FDA advisors said the trial investigators should continue the study, while keeping an eye out for further safety signals.
The Institute for Clinical and Economic Review flagged five drugs whose prices were raised in 2023 with no evidence to support it. Meanwhile, the makers of these drugs have been reporting double-digit sales growth for many of these products.
Incyte is abandoning its ALK2 blocker zilurgisertib, which it was trialing for myelofibrosis-associated anemia, while iTeos will deprioritize the development of inupadenant after it failed to meet the biotech’s clinical bar in a Phase II study of metastatic non-small cell lung cancer.
Ocaliva recently failed to secure the FDA’s traditional approval for primary biliary cholangitis due to safety concerns.
The company had said in October said that it was looking to license out its ex vivo editor renizgamglogene autogedtemcel to focus its resources on its in vivo platform.
The EMA approved a kidney disease–related label expansion for the blockbuster GLP-1 drug after a study showed reduced risk of death by 20%.
Pfizer, facing increasing pressure from Novartis, is touting a Phase III win for Ibrance as the first clinical evidence supporting the CDK4/6 inhibitor class’ use in patients with a specific type of breast cancer.
When hiring job candidates to work on cell and gene therapies, companies look for more than just technical skills. Talent acquisition executives from Bristol Myers Squibb and Intellia Therapeutics offer an inside look at what they want in an employee.