First Four Members Of US FDA’s Newest Advisory Committee Are Mostly Rookies

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.