The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to the investigational chimeric antigen receptor (CAR) T-cell therapy CT053, a fully human anti-B-cell maturation antigen autologous CAR T-cell therapy, for the treatment of relapsed/refractory multiple myeloma (MM).
Responses observed with CT053
The decision for RMAT designation was based on data from an ongoing phase I study in heavily pretreated patients with MM in China. So far, 19 of 24 patients with relapsed/refractory MM had a complete response to therapy, with no cases of grade 3/4 cytokine release syndrome observed during the study. Further data from this trial will be presented at the upcoming American Society of Hematology 2019 Annual Meeting.
RMAT designation is a program designed to expedite the drug development and review processes for promising regenerative medicines and advanced therapies, including CAR T-cell therapies. The designation is similar to that of fast track and breakthrough therapy designations, providing the ability for early interactions with FDA senior management to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application, and other opportunities to expedite development and review.
To date, 44 agents have received RMAT designation.