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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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Rep. Jake Auchincloss of Massachusetts said the Commissioner’s National Priority Voucher program did not receive congressional backing. The FDA has also not yet made disclosures for eight senior reviewers, according to Auchincloss.
Mounjaro and Zepbound combined for $11.7 billion in the fourth quarter, which beat analyst consensus of $10.6 billion.
Novartis will still be on the lookout for early-stage deals under $2 billion, and later-stage agreements around a product that could reach the market within five years, CEO Vas Narasimhan said Wednesday.
Pfizer announces the first data from its Metsera-acquired pipeline just ahead of its earnings call, where analysts pressed execs for more details; Merck and Roche also released Q4 and full year earnings, with Eli Lilly, Novo Nordisk and others reporting Wednesday; REGENXBIO hits a regulatory snag ahead of its upcoming PDUFA; more.
Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like MPS II moving into the spotlight.
Nearly 100 biotechs went public amid the industry’s IPO frenzy in 2021, driven by an influx of pandemic-driven investments. But many of those companies have little to show investors.
U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare pediatric disease priority review voucher program, among other initiatives, while ending a three-day partial government shutdown.
Novo Nordisk beat analyst expectations for the fourth quarter, but the result was overshadowed by softened expectations for this year.
Analysts parsed the limited data available for Pfizer’s obesity candidate on the pharma’s fourth-quarter earnings call Tuesday, looking for any nugget of additional context.
In its 2025 full year and fourth-quarter earnings call, Merck executives touted the merits of recent deals and what CEO Robert Davis called “probably the broadest and widest pipeline we’ve had in years.”