FDA grants fast track, RMAT designations to bispecific CAR-T for non-Hodgkin lymphoma
The FDA granted fast track and regenerative medicine advanced therapy designations to C-CAR039, a chimeric antigen receptor T-cell therapy in development for treatment of relapsed or refractory diffuse large B cell lymphoma.
C-CAR039 (Cellular Biomedicine Group, CBMG) is an autologous, gene-edited, bispecific CAR T-cell therapy that targets both CD19 and CD20 proteins on the surface of cancer cells.
The FDA's fast track designation helps to expedite development, review and potential approval of treatments for serious or life-threatening diseases.
The regenerative medicine advanced therapy (RMAT) designation program, part of the 21st Century Cures Act, is designed to expedite review of regenerative medicine therapies intended to treat, modify, reverse or cure serious or life-threatening diseases or conditions. Preliminary clinical evidence must indicate the therapy has the potential to address unmet medical needs.
The FDA based the fast track and RMAT designations on early results from an ongoing phase 1, investigator-initiated, multicenter trial being conducted in China. The study is designed to evaluate the safety and efficacy of C-CAR039 for patients with relapsed or refractory B-cell non-Hodgkin lymphoma.
Manufacturer-provided results showed an overall response rate of 92.6% among 28 patients eligible for safety analyses and 27 eligible for efficacy analyses. Researchers reported a complete response rate of 85.2%, and 74.1% of patients remained in complete remission at median follow-up of 7 months.
Most patients (96%) developed cytokine release syndrome. One patient developed grade 3 CRS; all other cases were grade 1 or grade 2.
Two patients experienced grade 1 immune effector cell-associated neurotoxicity syndrome.
"This is great news for CBMG that the FDA has granted C-CAR039 both RMAT and fast track designations based on its potential to increase objective and complete response rates in [patients with] relapsed or refractory DLBCL,” Tony (Bizuo) Liu, chairman and CEO of CBMG, said in a company-issued press release. “The clinical data based on our clinical trials in China continue to support the hypothesis that C-CAR039 is the best-in-class CAR-T asset for patients in this indication.”
CBMG will work with the FDA to initiate a phase 1b/phase 2 study of C-CAR039 in the U.S., Liu added.
The FDA previously granted orphan drug designation to C-CAR039 for the treatment of patients with follicular lymphoma.