FDA Grants Advanced Regenerative Medicine Therapy and Accelerated Designations to Novel CAR T-Cell Therapy for Relapsed and Refractory B-Cell Non-Hodgkin’s Lymphoma

C-CAR039 has shown positive efficacy and safety data in patients with relapsed or refractory B-cell non-Hodgkin’s lymphoma.

FDA officials have granted both Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation to C-CAR039, a novel autologous chimeric antigen receptor (CAR) bispecific T cell therapy, for the treatment patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL).

C-CAR039 targets both CD19 and CD20 antigens, and early results from an investigator-led trial demonstrate positive efficacy and safety data in patients with r/r B-cell non-Hodgkin lymphoma. As of April 20, 2021, 34 patients had received the treatment, of which 28 were eligible for safety analyzes and 27 were evaluable for efficacy analyses. The median age of patients was 55.5 years and 75% had stage 3/4 Ann Arbor cancer. Participants had a median of 3 prior lines of therapy, and transition therapy was given to 17.9% of patients.

According to a press release, the best overall response rate was 92.6%, with a complete response rate of 85.2%. Patients had a median time to response of 1 month and at a median follow-up of 7 months, 74.1% of patients were still in complete remission. In addition, the estimated progression-free survival rate at 6 months was 83.2%.

“It is great news for CBMG that the FDA has granted C-CAR039 RMAT and fast-track designations based on its potential to increase objective and complete response rate in DLBCL r/r,” said Tony Liu. , Chairman and CEO of Cellular Biomedicine Group. , in the press release. “Clinical data based on our clinical trials in China continue to support the hypothesis that C-CAR039 is the best-in-class CAR T active for patients in this indication.”

Cytokine release syndrome (CRS) was reported in 96% of patients, of which 92% were grade 1/2. Only 1 patient had grade 3 CRS. Immune effector cell-associated neurotoxicity syndrome occurred at grade 1 in 2 patients, and no grade 2 or higher neurological events were reported, according to the press release. Researchers will continue to evaluate patients with longer follow-up.

Additionally, the FDA’s Office of Orphan Product Development granted Orphan Drug Designation to C-CAR039 for the treatment of follicular lymphoma in June 2021. The Investigational New Drug Application was approved by the FDA on December 10, 2021 .

“We are working to initiate 1b/2 trials for C-CAR039 in the United States soon,” Liu said in the press release. “And we will work closely with the FDA to find the best way forward to get the drug to patients in the US and EU.”


CBMG receives Advanced Therapy and Accelerated Regenerative Medicine designations from the FDA. Press release. CBMG; January 12, 2022. Accessed January 13, 2022. https://www.cellbiomedgroup.com/newsroom/fda-rmat?lang=en