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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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Most respondents expressed concerns about the economic environment, as competition for jobs jumped more than 130%.
In the biopharma industry, engineers are pivotal drivers of innovation, helping to shape the landscape of healthcare discoveries.
While the FDA considers a T cell malignancy risk to be applicable to all commercial CAR-T therapies, Gilead Sciences’ Tecartus has a revised warning that potential adverse events “may” occur.
The regulator’s Complete Response Letter flagged issues with the new formulation of tesamorelin’s chemistry, manufacturing and controls and sought more information about its immunogenicity risk.
While decentralized trials are expediting innovation and promoting diversity, challenges remain, including complex stakeholder ecosystems, skills gaps and regulation.
The company said it’s preparing to file a New Drug Application with the FDA on the strength of late-stage study data for its investigational RNA-targeted donidalorsen in patients with hereditary angioedema.
An FDA advisory committee will meet to review J&J and Legend Biotech’s supplemental BLA for Carvykti for the treatment of relapsed or refractory multiple myeloma patients who have undergone at least one prior line of therapy.
In a Phase I/II trial, an 11-year-old boy gradually regained hearing—eventually reaching normal range for some frequencies at 30 days—following treatment with Eli Lilly’s dual adeno-associated viral vector-delivered gene therapy.
Drug discovery requires complex and multi-faceted decisions to justify billion-dollar investments that will take years to realize.
This week, Lori, Greg and Tyler discuss the first surge of IPO activity this year plus gene therapy pricing,