FDA
Jubilant HollisterStier received a warning letter over problems that the FDA said necessitate an immediate and comprehensive assessment of the contract manufacturing organization’s global operations.
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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?
DemeRx is launching the first U.S. clinical trial of an ibogaine-derived drug candidate, marking a pivotal moment for a controversial psychedelic long sidelined by safety concerns.
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Among the health department’s efforts is an expedited investigational new drug pilot program that would leverage collaborations with U.S. research institutions to reduce early trial timelines by as much as 12 months.
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.
The FDA in February briefly refused to review Moderna’s flu vaccine, citing trial inadequacies, but reversed course a few days later. A verdict is expected by Aug. 5.
After being rejected in June 2022, GSK paid Spero Therapeutics $66 million for an exclusive worldwide license to develop and commercialize the drug. It’s the second oral antibiotic GSK has brought to market since the beginning of last year.
After a regulatory odyssey that delayed a filing for what would be the first genetic medicine for Huntington’s disease, the FDA has agreed that three-year data from uniQure’s Phase 1/2 trial are sufficient to support an accelerated biologics license application.
Moderna appears to have aligned with the FDA ahead of an advisory committee meeting for its mRNA-based flu vaccine, which the regulator initially turned away in February; biotech IPOs are going gangbusters, including two new records raises in as many weeks; layoffs continue across biopharma; plus much more.
The Vaccines and Related Biological Products Advisory Committee will meet June 18 to discuss Moderna’s seasonal flu vaccine mRNA-1010 after the FDA initially refused to accept the application in February.
Sanofi makes no mention of the Commissioner’s National Priority Voucher. Tzield was awarded the ticket in October 2025, but Sanofi requested withdrawal from the program after former CDER head Tracy Beth Høeg reportedly expressed skepticism of the drug.
The FDA rejected bitopertin in February amid reports of skepticism from former CBER director Vinay Prasad, who has since departed the agency.
The FDA has expanded the regulatory toolkit during President Donald Trump’s second term, adding new mechanisms for rare diseases while putting the Biden-era platform technology designation into action.