FDA grants Regenerative Medicine Advanced Therapy (RMAT) designation for CAR-T cell therapy, CT053

On the 28^th of October 2019, 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, for the treatment of patients with relapsed/refractory (R/R) multiple myeloma (MM).¹

CT053 is a fully human, autologous CAR T-cell therapy against B Cell Maturation Antigen (BCMA), a protein commonly overexpressed in MM, involved in the regulation of B cell proliferation and survival, as well as plasma cell maturation and differentiation. The approval is based on clinical data of an ongoing, three-site Phase I/II trial (NCT03975907) in 84 heavily pre-treated patients with R/R MM. The updated clinical data on efficacy and safety will be presented during the 61^st American Society of Hematology meeting in December this year.

The drug has previously received Orphan Drug designation from the FDA (read here) and priority medicines (PRIME) eligibility from the European Medicines Agency (EMA).