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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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With the FDA’s approval on Thursday, Dupixent can now be used for the treatment of children aged 1 to 11 years who have eosinophilic esophagitis, a chronic disease that can severely impact their ability to eat.
Ahead of a Jan. 31 vote, Senate health committee chair Sen. Bernie Sanders on Thursday said he has the votes needed to subpoena the CEOs of Johnson & Johnson and Merck to testify on drug pricing.
Despite myriad stumbling blocks over the past two years, experts still see the potential of Bruton’s tyrosine kinase inhibitors to quiet progressive multiple sclerosis.
In the CONTACT-02 pivotal trial, the combination of Exelixis’ Cabometyx and Roche’s Tecentriq reduced the risk of disease progression or death by 35% in patients with metastatic castration-resistant prostate cancer.
After missing its Jan. 24 PDUFA date, the regulator has extended its review period but has not issued a new decision timeframe or requested additional data, Liquidia announced Thursday.
BioSpace’s Lori Ellis and Chantal Dresner discuss anticipated job market trends for 2024 including unemployment, anticipated job search activity and hiring trends.
The bladder cancer-focused company’s larger-than-expected Nasdaq debut on Thursday opens this year’s batch of planned initial public offerings, which include at least half a dozen biotechs.
More than 3,000 people in the U.S. suffered from side effects of Ozempic in 2023, according to exclusive reporting on Wednesday from Reuters citing data from America’s Poison Centers.
After 26 weeks of follow-up, five of six children treated with an experimental gene therapy developed in China showed strong improvements in speech perception and could hold normal conversations.
The FDA’s Peter Marks and Nicole Verdun in the NEJM Wednesday disclosed that more than 27,000 CAR-T doses have been administered in the U.S. as of the end of 2023, of which there are 22 cases of T-cell cancers.