FDA Grants Fast Track Designation to C-CAR039 for DLBCL


The FDA granted both a regenerative medicine advanced therapy and fast track designation to C-CAR039 for relapsed or refractory diffuse large B-cell lymphoma.

The FDA has granted both a fast-track designation and regenerative medicine advanced therapy designation to the autologous bi-specific CAR-T therapy that targets both CD19 and CD20, C-CAR039, for the treatment of relapsed or refractory diffuse large B cell lymphoma (DLBCL), according to a press release by Cellular Biomedicine Group Inc.1

"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 r/r DLBCL. 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,” said Tony (Bizuo) Liu, chairman and chief executive officer, in a press release.

The agent was previously granted clearance for clinical development by the FDA for the treatment of relapsed or refractory non-Hodgkin lymphoma.

The clearance granted phase 1b clinical development of the agent. C-CAR039 has also been granted an orphan drug designation for the treatment of follicular lymphoma. A study of the agent conducted in China found that it had a promising efficacy and safety profile.2

“We are excited about the potential of C-CAR039 as a best-in-class treatment in r/r B-NHL. C-CAR039 clearly showed potential superior efficacy and favorable safety results for patients with r/r B-cell NHL. Our C-CAR039 trial is a testament of the teams’ translational medicine capability. We look forward to bringing more potentially promising development to clinical trials,” said Yihong Yao, PhD, chief scientific officer of CBMG, in a press release.

As of April 2021, 34 patients had received C-CAR039, with 28 patients being evaluable for safety and 27 for efficacy. The median age of patients was 55.5 years, and 75% had cancer of Ann Arbor stage III/IV. The median number prior lines of therapy was 3. Nearly 18% of patients had received bridging therapy.

The best overall response rate was reported at 92.6%, with a complete response of 85.2%. The median time to response was 1 month. At a median follow up of 7 months, 74.1% remained in complete remission. The estimated 6-month progression-free survival was at 83.2% (95% CI, 69.1-100.0).

In terms of safety, 96% experienced cytokine release syndrome, with 92% of cases being grade 1 or 2. Only 1 patient experienced grade 3 cytokine release syndrome. All immune-effector cell-associated neurotoxicity syndrome were grade 1 or 2. No grade 3 or higher neurological events were reported.

Longer follow-up studies of C-CAR039 are currently planned, according to Liu.1 

“We are working towards initiating 1b/2 trials for C-CAR039 in the US soon. 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," he stated, in a press release.

1 CBMG receives FDA regenerative medicine advanced therapy and fast track designations for bi-specific anti-CD19/CD20 CAR-T cell therapy for relapsed/refractory B-cell non-Hodgkin lymphoma. News release. CBGM. January 12, 2022. Accessed January 12, 2022.
2 CBMG receives FDA clearance of IND application for bi-specific anti-CD19/CD20 CAR-T cell therapy for relapsed/refractory B-cell non-hodgkin lymphoma. News release. CBGM. December 13, 2021. Accessed January 12, 2022. https://bit.ly/3fgaCaW.
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