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.

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

The Japan-based drug maker is hoping to expand into cell and gene therapy as well as enlarge its presence in the North American market, while Orchard awaits an FDA verdict on Libmeldy.