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.

Despite promising clinical data and solid regulatory plans, biotech firms hoping to address the unmet need in Sanfilippo Syndrome through gene therapy are facing financial struggles.