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.

Private Company Edition: Quarter-over-quarter increases in venture capital have been bolstered by fewer but larger rounds, like the recent $245m series A for Aiolos. Also, OrbiMed raised $4.3bn across three new VC funds, Rampart completed an $85m series A and Atom Bioscience raised an $83m series D. 

Institute for Clinical and Economic Review continues to assign high values to novel gene therapies, suggesting that Orchard’s pending ‘arsa-cel’ for MLD could be cost effective at a price as high as $4 million per treatment.

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