In 2020, the FDA approved the third CAR-T cell treatment for cancer, Tecartus to treat mantle cell lymphoma, but it also sent multiple developers of gene and cell therapies back to the drawing board—demanding additional data to support approval of their products. These high-profile rejections signaled a new approach to the regulatory review of gene and cell therapies.
The FDA has hired more than 50 new reviewers in anticipation of receiving 200 applications per year from companies hoping to start clinical trials of gene and cell therapies and issuing 10 to 20 new approvals a year by 2025. This represents both an opportunity and great challenge for gene and cell therapy developers, who will need to learn how to navigate the regulatory process effectively.
The potential impact of regulatory surprises on biopharma developers and their investors was perhaps best demonstrated by Biomarin. After years of developing a gene therapy to treat hemophilia, it saw its shares plummet in August 2020 on news that the FDA refused to approve the product without an additional two years’ worth of data on its durability.
We’ll review the latest developments in the regulatory review process for gene and cell therapies, offering advice that companies can use to streamline their interactions with regulators, both in the U.S. and overseas.
- Understanding the FDA’s new guidance documents covering product development, accelerated approval pathways and other topics specific to gene and cell therapies.
- How to apply the lessons learned from Biomarin’s setback and other approval delays to improving development strategies for future gene and cell therapies.
- Comparing and contrasting the FDA’s policies for gene and cell therapies to those of regulatory bodies in other large global markets.