FDA Grants Regenerative Medicine Advanced Therapy, Fast Track Designations to Novel CAR T-Cell Therapy for Relapsed, Refractory B-cell Non-Hodgkin Lymphoma


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

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

C-CAR039 targets both the 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 received the therapy, 28 of whom were eligible for safety analyses and 27 of whom were evaluable for efficacy analyses. Patients’ median age was 55.5 years and 75% had cancer of Ann Arbor stage 3/4. Participants had a median of 3 prior lines of therapy and bridging therapy had been 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. Furthermore, the 6-month estimated progression-free survival rate was 83.2%.

“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 rate in r/r DLBCL,” said Tony Liu, chairman and CEO of Cellular Biomedicine Group, in the 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.”

Cytokine release syndrome (CRS) was reported in 96% of patients, 92% of which was 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 neurologic events were reported, according to the press release. The researchers will continue to evaluate patients with longer follow-up.

Separately, the FDA Office of Orphan Products Development granted an Orphan Drug Designation to C-CAR039 for the treatment of follicular lymphoma in June 2021. The Investigational New Drug application was cleared by the FDA on December 10, 2021.

“We are working toward initiating 1b/2 trials for C-CAR039 in the US soon,” Liu said in the press release. “And we will work closely with the FDA to seek the best path forward to deliver the drug to patients in the US and EU.”


CBMG Receives FDA Regenerative Medicine Advanced Therapy and Fast Track Designations. News release. CBMG; January 12, 2022. Accessed January 13, 2022. https://www.cellbiomedgroup.com/newsroom/fda-rmat?lang=en

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