Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.
Rare disease advocates want Congress to initiate a process that could result in a formal definition for an ultra-rare disease and additional incentives for drug development in the space.
Among the legislative asks that the EveryLife Foundation for Rare Diseases encouraged participants to push during a 2 March Capitol...
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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