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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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Replimune is axing 144 employees at its Woburn headquarters and 80 at its Framingham manufacturing site. The cuts follow the FDA’s second rejection of the biotech’s advanced melanoma candidate.
With programs from Eisai and Hansoh Pharmaceutical in hand, Tortugas Neuroscience has emerged with hopes of delivering daily oral treatments to patients with central nervous system conditions.
A triplet regimen comprising Merck’s Welireg and PD-1 blockbuster Keytruda and Eisai’s Lenvima flopped in a Phase 3 renal cell carcinoma trial.
Moderna, Revolution Medicines and Zymeworks record wins in melanoma, lung and breast cancers, while BeOne swings and misses at head and neck cancer.
The Phase 3 failure has prompted Gilead Sciences and Arcus Biosciences to terminate a mid-stage study of their TIGIT asset in lung cancer.
At Sarepta Therapeutics, we’ve seen it all. Here are the questions I believe we should be asking to move forward in Duchenne muscular dystrophy.
Viatris, which Pfizer created in 2020, voluntarily withdrew extended-release products made at a plant in Ireland after an analysis revealed an issue that could affect bioavailability.
Doubling survival in pancreatic cancer, a long-fought rare disease approval, a massive IPO and ambitious biotech entrepreneurs have BioSpace Senior Editor Annalee Armstrong feeling upbeat about the biotech scene.
After entering the CAR T space in February, Eli Lilly is “jumping into in vivo CAR-T with both feet” with the acquisition of Kelonia Therapeutics and its gene delivery technology.
An investigational cocktail was tied to a 0% overall response rate in patients with gastroesophageal cancer, but developers Agenus and MiNK Therapeutics aren’t giving up on the program just yet.