Agency recommendation for randomized controlled trials in ultra-rare pediatric disease is unworkable for various reasons, say advocates and sponsors who seek use of the accelerated approval pathway based on changes in heparan sulfate levels.

Only two breakthrough-designated novel agents remain on the US FDA drugs center’s 2023 user fee calendar, while CBER stacks up on vaccines and gene therapies under both BTD and RMAT programs.

Highlights of recent submissions for approval from the Pink Sheet’s US FDA Performance Tracker

Emerging Company Profile: The US biotech is developing a series of recombinant fusion proteins to tackle alopecia and other diseases without supressing the immune system.