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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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Merck is eyeing a quick review for its lipid-lowering drug candidate enlicitide, which in December was awarded a Commissioner’s National Priority Voucher.
Serial biotech investor Kevin Tang previously unsuccessfully tried to buy Kezar Life Sciences via Concentra Biosciences, a biotech consolidation company owned by the venture capital firm he runs.
Takeda’s layoffs include cutting 247 people in Massachusetts. The workforce reduction is meant to help offset investments in areas including a product launch for oral drug candidate zasocitinib, for which the pharma today announced positive Phase 3 data.
While tonlamarsen missed one of two co-primary endpoints, Kardigan says the drug has shown a clinically meaningful effect on blood pressure, supporting advancement into Phase 2b.
The FDA rejected the high-dose regimen of Spinraza in September last year due to manufacturing concerns.
William Blair hailed a positive readout in cutaneous lupus erythematosus as a turning point for Biogen, while RBC Capital analysts called the results “another derisking step” for the company’s immunology and inflammation pipeline.
The lack of a dose-response effect could be due to the high number of dropouts in the higher-dose Winrevair arm and the relatively small study population, a discussant for Merck explained.
The Insilico Medicine agreement plays into Eli Lilly’s recent AI push, anchored by a partnership last year with NVIDIA to build a supercomputer to optimize drug discovery and shorten the development timeline.
AbbVie’s Skyrizi appears to have stronger efficacy than Johnson & Johnson’s newly approved pill Icotyde, as well as a less frequent dosing schedule that patients could prefer, according to analysts at BMO Capital Markets.
The FDA has some big verdicts lined up in the second quarter, including one for a closely watched obesity drug that many anticipate will further intensify competition in weight loss.