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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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While a substantial portion of pipeline assets are externally sourced, many Big Pharmas are tapping into incubators and venture funds to uncover cutting-edge scientific trends, determine their future focus points and even carve out a niche in an emerging geographical hotspot.
Albert Bourla confirmed that he called President Donald Trump after receiving a letter asking Pfizer and a clutch of other pharmaceutical companies to lower drug prices or face consequences.
BMN 390 fell short of an immunogenicity threshold that BioMarin was looking for to support its further development. Employees working on the program have been redeployed within the company.
The number of biopharma professionals let go has increased year over year for three straight months. In July, as many as 8,000 people lost or were projected to lose their jobs, due largely to news that Merck projects to cut roughly 6,000 employees as part of a multiyear process.
Vertex reported healthy revenue in its second quarter earnings report, though news of VX-993’s mid-stage trial results and lack of alignment with the FDA regarding an expanded label for Journavx tempered analyst reactions, sending the stock down 13%.
Praxis’ vormatrigine reduced seizures by 56.3%, an effect size that, according to analysts at Truist Securities, exceeds that of its closest competitors.
While the deaths occurred in patients who had been treated with Agios’ anemia treatment Pyrukynd, the biotech insisted in an SEC filing midday Monday that the drug’s risk-benefit profile remains unchanged.
In an open letter, 22 experts who designed and ran Replimune’s Phase III IGNYTE trial answered the FDA’s issues, as outlined in the complete response letter for the melanoma candidate RP1.
From innovation in manufacturing to more-flexible regulation and better communication with payers, much needs to happen to make CGTs commercially viable. But it is possible, experts agreed at a recent panel.
The primary focus in scaling up production should first be the adoption of lean manufacturing principles used in virtually every other industry.