FDA
Viridian Therapeutics’ Lumvoa is the first FDA-approved treatment for thyroid eye disease that includes data for both active and chronic forms of the illness.
FEATURED STORIES
The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven.
The FDA has so far secured 600 new hires and is looking for 1,600 more as interim leadership at the agency aims to rebuild the workforce and morale after more than a year of intense attrition.
Former FDA, CDC and NIH leaders convene at the BIO International Convention to discuss the dismantling of the Department of Health and Human Services under the Trump administration—and where we go from here.
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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.
A few short days after announcing an FDA pivot on a separate asset, REGENXBIO is planning to test the agency’s apparent newfound rare disease outlook on another late-stage gene therapy.
A new report from RBC Capital Markets lists Biotechnology Innovation Organization chief executive John Crowley and military physician Heidi Overton as potential candidates for the role of FDA commissioner—in addition to the agency’s current acting leader, top food regulator Kyle Diamantas.
AbbVie scooped up immunology player Apogee Therapeutics for nearly $11 billion in one of the year’s top deals to-date, while Sanofi made a big play to survive its upcoming Dupixent patent cliff; FDA uncertainty continues as the agency changes direction on gene therapies by uniQure and REGENXBIO; and Jef Akst and Annalee Armstrong report back from San Diego.
As Sangamo runs out of cash, Eli Lilly and Astellas have emerged as stalking horse bidders for key assets, including a Fabry gene therapy currently being submitted for potential FDA approval.
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.
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?