Not everyone reviewing Daiichi Sankyo’s EU marketing application for Turalio agreed that the potential tenosynovial giant cell tumor treatment shouldn't be approved.
Worries about unpredictable, potentially life-threatening effects of an orphan drug for treating a non-fatal condition. Missing data and uncertainty over the efficacy of the product even though it met its primary endpoint in a Phase III trial. A decision in the EU that contrasts sharply with the viewpoint in the US.
The statements all relate to Turalio (pexidartinib), Daiichi Sankyo’s drug for tenosynovial giant cell tumor (TGCT), and the European Medicines...
Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.
The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.
Under current law, drugs or biologics must receive rare pediatric disease designation before 1 October to be eligible for a priority review voucher. The sunset provisions have led to a spike in designation requests at the FDA and a push by pediatric and rare disease advocates for an extension, either through a continuing resolution or reauthorizing legislation.
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
Revised recommendation is based on most recent data on circulating strains in US, where KP.3 is now dominant. Novavax sticks with plans targeting the JN.1 ‘parent strain.’
Pink Sheet reporter and editors discuss the US FDA’s changes to the COVID-19 vaccine indication and its revocation of the vaccine emergency use authorizations, as well as new vaccine coverage and reimbursement questions.
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