The FDA has granted its regenerative medicine advanced therapy designation to CT053, a chimeric antigen receptor T-cell therapy for the treatment of patients with relapsed or refractory multiple myeloma.
CT053 (CARsgen Therapeutics) is an investigational, fully human, autologous anti-B-cell maturation antigen CAR T-cell therapy that targets BCMA on the surface of cancer cells.
"The RMAT designation indicates that CT053 has demonstrated potential to address unmet medical needs for patients with [relapsed or refractory multiple myeloma],” Zonghai Li, MD, PhD, CEO of CARsgen said in a press release.
“The designation is a remarkable achievement towards expediting the product development and review of our planned biologics license application (BLA) and will be invaluable to bringing this cutting-edge advance to patients as quickly as possible,” Li added.
The RMAT designation was awarded based on data from an ongoing phase 1 clinical trial of patients in China with heavily pretreated multiple myeloma. Updated clinical trial data will be presented during this year’s American Society of Hematology annual meeting in Orlando, according to the press release.
The FDA previously granted CT053 with its orphan drug status for the treatment of multiple myeloma. Results from an exploratory phase 1 clinical trial in China showed that 19 of 24 patients with relapsed or refractory multiple myeloma had a complete response to therapy, with no cases of high-grade (3 or 4) cytokine release syndrome during the study.
The FDA’s RMAT designation program is part of the 21st Century Cures Act and was created to expedite regenerative medicine therapies — defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations — that is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition. The therapy must have preliminary clinical evidence indicating the drug has the potential to address unmet medical needs for such a disease or condition.