To date, the FDA has given the green light to 12 gene therapies, and the agency’s top drug regulator aims to pave the way for more approvals in the future.
During a webinar hosted by the Alliance for a Stronger FDA, a lobbying organization for the agency, Peter Marks, M.D., who heads the FDA’s Center for Biologics Evaluations and Research (CBER), stated that if the agency only approves two or three gene therapies annually over the next few years, it would be considered a failure.
Peter Marks remarked that achieving success in terms of gene therapy approvals would involve a logarithmic or exponential increase in the number of therapies approved over time. Currently, the FDA has given the green light to 12 gene therapies, with five of those being approved in 2022.
Marks and CBER have outlined a four-point plan to achieve this goal, which includes enhancing manufacturing processes through research, providing more clarity on the use of the accelerated approval pathway for gene therapies, ensuring coordinated regulatory decisions with other agencies, and launching an Operation Warp Speed-like initiative for rare diseases. However, implementing these strategies may not be straightforward.