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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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After greenlighting 56 novel therapeutics in 2025, four notable applications continue to await the agency’s action after being delayed from the fourth quarter last year.
After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told BioSpace, with both rare and chronic diseases headlining investor and R&D interest as JPM26 kicks off.
Insmed’s freshly approved lung condition drug soared past sales expectations for 2025, netting $144.6 million in its first full quarter of sales.
Roche first partnered with MediLink in January 2024, likewise for an antibody-drug conjugate for solid tumors.
Rampart Bioscience was working on a platform to deliver gene therapies without the need for viral vectors.
Pharmas will need to provide their latest stance on the Most Favored Nation drug pricing plans, while biotech finally gets a break after a few tough years.
Aside from the layoffs, InflaRx will deprioritize Gohibic, a COVID-19 antibody that was granted emergency use authorization in 2023. The therapy failed a late-stage trial in a rare skin disease last year.
Rumors of a Revolution buyout come as the industry gears up for one of its biggest trade conferences, where observers expect many such deals to be announced.
The DC-based biopharma disputed the FDA’s conclusions regarding the data provided in its supplemental application for Hetlioz and promised to keep pushing for an approval.
Eli Lilly has expressed interest in participating in the IPO, with the regulatory filing revealing the obesity juggernaut’s plans to buy as much as $100 million worth of Aktis shares.