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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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The Connecticut-based biotech, which emerged from stealth last year, has secured $202 million to date as it looks to move two assets targeting prostate and breast cancer into the clinic.
FibroGen expects its headcount reduction, which is tied to eliminating 75% of its U.S. workforce, to be mostly complete by the end of the first quarter of 2025.
Ascendis Pharma’s palopegteriparatide was previously rejected by the regulator due to manufacturing problems, and the review period for its resubmitted application was extended by three months.
A day after the regulator denied approval of Lykos’ MDMA-assisted PTSD therapy, a medical journal pulled three studies related to the use of the psychedelic in the indication.
Humacyte’s stock price dropped 18% in after-hours trading Friday after the company announced the FDA requires additional time to review its Biologic License Application. No reason was given for the delay or an updated decision date.
ARS Pharma’s neffy on Friday became the first FDA-approved nasal spray to address severe allergic reactions, including those that might lead to life-threatening anaphylaxis.
Hundreds of companies are currently running clinical trials in the increasingly lucrative obesity space. BioSpace looks at five candidates with data expected before the end of the year.
In the next two weeks, the FDA will hand down its verdicts for three drug applications, including ones for multiple myeloma and hypoparathyroidism therapies.
Clinical trial concerns and a negative advisory committee vote ultimately sunk the treatment.
AN2 is discontinuing a study that evaluated epetraborole for treatment-refractory MAC lung disease and plans to shift focus to its boron chemistry platform.