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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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Hit with lower sales of its COVID-19 antiviral Veklury and the weak performance of its HIV franchise, Gilead Sciences reported a 4% year-over-year revenue loss in the fourth quarter of 2023.
Thanks to a rough launch of its Biogen-partnered Alzheimer’s disease treatment, Eisai will likely miss its target of treating 10,000 patients with Leqembi by the end of March 2024.
With manufacturing issues persisting, last year’s shortages of medicines, including chemotherapies, weight-loss drugs and antibiotics, will continue into 2024.
Politics aside, both the government and the pharmaceutical industry want to bring affordable effective therapies to patients. Implementation is the obstacle. Working together is the only way to modify the IRA to do what it intends to do: benefit patients.
The monoclonal antibody Nipocalimab, which J&J acquired from Momenta Therapeutics, met its primary endpoints in mid-stage and late-stage trials in myasthenia gravis and Sjögren’s disease.
The French vaccine maker on Monday said it sold a priority review voucher, which was awarded by the FDA in November 2023 alongside an approval for its chikungunya vaccine, to an undisclosed buyer.
Design patents, trademarks, copyright and trade secrets offer lower cost, and sometimes underappreciated, forms of intellectual property.
Eli Lilly on Tuesday reported more than $9.3 billion in revenue in the fourth quarter of 2023, beating Wall Street expectations, thanks to demand for its weight-loss drug Zepbound and diabetes treatment Mounjaro.
The company’s three-drug combination met all primary and key secondary endpoints in two randomized controlled trials in cystic fibrosis patients, paving the way for an application to regulators.
The antibody-drug conjugate, in combination with Takeda’s Velcade and the steroid dexamethasone, reduced the risk of death and disease progression by 59% versus a similar Darzalex-based regimen.