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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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The new CEO has at least five years and a large M&A war chest to position Sanofi for life after the loss of exclusivity on its cornerstone immunology product.
While tovecimig met the main goal of progression-free survival in a Phase 2/3 trial, it did not improve overall survival.
Sun Pharmaceutical is bringing Merck spinoff Organon into the fold, paying $11.75 billion in hopes of becoming a top global biosimilar player.
Eli Lilly is picking up Ajax Therapeutics and its once-daily oral blood cancer candidate. The deal follows the pharma’s buyouts of ADC specialist CrossBridge Bio and in vivo CAR T company Kelonia Therapeutics.
With Phase 3 data in hand, Intellia Therapeutics is seeking approval for its in vivo CRISPR gene editing therapy for hereditary angioedema.
FDA inspectors warned UCSF radiopharmaceutical facility that losing environmental control could pose a serious hazard to patients.
George Church’s Rejuvenate Bio is turning to social networks to help fund its work on one-time gene therapies targeting chronic diseases and root causes of aging.
After striking a Most Favored Nation deal with the White House in January, Johnson & Johnson will now offer Xarelto at 68% off on TrumpRx, dropping its price from $611.82 to $197 per 30-pill pack.
As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” session focused on the need to expedite more bespoke gene editing treatments like the one that saved young KJ Muldoon.
While the FDA did not announce the recipient names of the Commissioner’s National Priority Vouchers, the agency’s descriptions of the awarded products match those in development at Compass Pathways, Transcend Therapeutics and Usona Institute.