Immuno-Oncology Resource Center

Immuno-Oncology Resource Center


Press Release

December 31, 2020
2 min read

Manufacturers submit rolling BLA to FDA for CAR-T to treat advanced multiple myeloma


Press Release

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Janssen and Legend Biotech have begun a rolling submission of a biologics license application to the FDA for ciltacabtagene autoleucel to treat adults with relapsed or refractory multiple myeloma.

Ciltacabtagene autoleucel (JNJ-4528), also known as cilta-cel, is an autologous chimeric antigen receptor T-cell therapy that targets the B-cell maturation antigen (BCMA) on the surface of cancer cells. Cilta-cel comprises a 4-1BB costimulatory domain and two BCMA-targeting single-domain antibodies with a preferential CD8-postive T-cell expansion.

Bone marrow aspirate cytology of multiple myeloma, a type of bone marrow cancer of malignant plasma cells, associated with bone pain, bone fractures and anemia.
Source: Adobe Stock.

The therapy is being co-developed by Janssen and Legend Biotech after the two companies announced a worldwide license and collaboration agreement for cilta-cel in 2017.

“We are committed to innovation in cell therapy and advancing the science of multiple myeloma to improve patients’ lives,” Peter Lebowitz, MD, PhD, global therapeutic area head for oncology at Janssen Research & Development, said in a company-issued press release. “[This] milestone is the culmination of a remarkable clinical development effort and collaboration with Legend Biotech.”

The FDA previously granted cilta-cel breakthrough therapy designation and has agreed to a rolling review of the BLA. The process will allow the manufacturers to submit completed portions of the application for review on a rolling basis, according to statements from the manufacturers.

The BLA was based on results of the phase 1b/phase 2 CARTITUDE-1 trial, in which 97 patients (median age, 61 years; 58.8% men) with relapsed or refractory multiple myeloma received citla-cel. Initial results of the trial, presented at this year’s virtual ASH Annual Meeting and Exposition, showed an overall response rate of 96.9% and a stringent complete response rate of 67% at median follow-up of 12.4 months. Median PFS had not yet been reached.

Researchers reported a 12-month PFS rate of 76.6% (95% CI, 66-84.3) and a 12-month OS rate of 88.5% (95% CI, 80.2-93.5).

“Initiation of the BLA submission is an important milestone in advancing this therapy for patients with multiple myeloma who are heavily pretreated and in need of treatment options,” Ying Huang, PhD, CEO and CFO of Legend Biotech, said in a company-issued press release. “Together with our collaborator Janssen, we look forward to working with the FDA to fulfill this unmet medical need with the goal of making this breakthrough treatment available to patients and health care providers in the future.”


Madduri D, et al. Abstract 177. Presented at: ASH Annual Meeting and Exposition (virtual meeting); Dec. 5-8, 2020.