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The resubmission for RGX-121, expected in the third quarter, comes as the FDA has deemed REGENXBIO’s existing data “sufficient” to support an accelerated filing. It immediately follows a similar reversal of position regarding uniQure’s embattled Huntington’s disease gene therapy.
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If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
While drugmakers and other stakeholders want to see faster approvals, experts say the FDA’s Commissioner’s National Priority Voucher program is still bereft of important details, with candidate selection and interference from the agency’s senior leaders topping the list.
UniQure’s planned third-quarter submission for its Huntington’s disease gene therapy may be a harbinger of a more flexible FDA under acting commissioner Kyle Diamantas—but how long will it last? And how can companies be sure these positive decisions won’t just be reversed?
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FDA veteran Peter Marks will now shape the future of Eli Lilly’s vaccines work after the buys of Curevo, LimmaTech Biologics and Vaccine Company for up to $3.8 billion total.
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Daiichi Sankyo’s full-year report was originally scheduled for April 27 but has now been pushed back to May 11. That same day, the pharma expects to release its five-year business plan.
Pfizer’s decision to cut its early-stage cancer asset was due to “strategic business reasons” and not driven by safety or efficacy concerns.
Of the 17 companies that were implored by the White House last July to apply Most Favored Nation pricing to their drugs, Regeneron is the last to agree—the same day the FDA greenlit its gene therapy for hearing loss in kids.
The FDA is signaling change, but actual success depends on more than simply bringing in a new leader at the Center for Biologics Evaluation and Research; it requires accountability, transparency and consistent action.
Approved Thursday via the FDA’s Commissioner’s National Priority Voucher program, Otarmeni is the first gene therapy for hearing loss—and the first treatment to target an underlying cause of the condition.
The takeover will give Amneal control of four facilities to manufacture biosimilars for a planned wave of launches in the coming years.
Chief Scientific Officer Pedro Beltran will succeed Eli Wallace as CEO of BridgeBio Oncology Therapeutics, as the board eyes a busy period of clinical advancement in the RAS oncology space.
Sanofi’s interim leadership sought on a Thursday earnings call to quell concerns that its sudden defense of Dupixent’s patents had anything to do with the departure of CEO Paul Hudson.
The FDA in July 2025 made publicly available over 200 complete response letters—an initiative that the investment community sees as “unanimously positive,” analysts told BioSpace.