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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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Viridian Therapeutics’ elegrobart normalized the degree of eye protrusion and improved double vision in a Phase 3 study. The company plans to file for approval in the first quarter of 2027.
Johnson & Johnson plans to advance the co-antibody therapy, which combines its IL-23 blocker Tremfya and the TNF-alpha inhibitor Simponi, into late-stage testing for Crohn’s disease and ulcerative colitis.
Facing increasing pressure from both industry and the White House, FDA Commissioner Marty Makary said the strong bad press against him is “corporate spin” and that the agency has “followed the science.”
First quarter earnings continue to arrive, with analysts demanding more from cautious Pfizer and Eli Lilly expecting more revenue; the FDA taps Katherine Szarama as Vinay Prasad’s controversial FDA tenure ends; oncology veterans miss Richard Pazdur at the agency’s first adcomm in nine months; and QurAlis and Corcept Therapeutics spark renewed hope in ALS.
In Salt Lake City, biotech founders new and seasoned reflect on ways to ride out the industry’s challenges, such as sending cold emails to investors and learning to address leadership weaknesses.
A legal settlement has put wind behind Pfizer’s sales into 2029—at which point key obesity moves will take the helm.
The action affects BioNTech sites in Germany and Singapore, where the company expects to have excess capacity.
Biogen’s Qalsody won FDA approval in 2023 to treat a rare, genetic form of amyotrophic lateral sclerosis. On Tuesday, QurAlis presented interim Phase 2 data showing the potential of a similar drug to more broadly treat the neurodegenerative disease.
Despite the continued decline of Pfizer’s COVID-19 products, shares stayed stable on Tuesday morning after the New York pharma reported a first quarter beat, which was led by Eliquis and Ibrance.
Madrigal Pharmaceuticals has licensed from Arrowhead Pharmaceuticals an RNA interference candidate that targets a genetic mutation present in around 30% of patients with MASH.