Legend Biotech (a subsidiary of GenScript Biotech) has received approval from the China National Medical Products Administration (NMPA) for cell therapy product, ciltacabtagene autoleucel (cilta-cel).

The treatment is approved for use in adults with relapsed or refractory multiple myeloma (MM) who have undergone at least three prior lines of therapy, including at least one proteasome inhibitor and one immunomodulatory agent.

Cilta-cel is a gene-modified autologous chimeric antigen receptor T cell (CAR-T) therapy targeting B-cell maturation antigen (BCMA).

It features a unique CAR structure composed of two BCMA-targeting, heavy-chain, single-domain antibodies. This design allows cilta-cel to bind the BCMA-expressing myeloma cells, and induce activation and proliferation of T cells to eliminate tumour.

Legend Biotech CEO Ying Huang stated: “The approval of cilta-cel in the China market marks a key milestone and will bring significant benefits to many patients. Moving forward, we will continue to pursue our goal of curing patients, expand our clinical research, and enhance the accessibility of this innovative product to benefit more patients.”

The approval is based on results from the CARTIFAN-1 Phase II trial, which evaluated the efficacy and safety of cilta-cel.

Results from a median follow-up of 37.29 months showed that among 58 patients analysed for efficacy, the overall response rate (ORR) was 87.9%. A very good partial response (VGPR) or better was achieved in 86.2%, and complete response (CR) or stringent complete response (sCR) was reached in 79.3%.

The median duration of response (mDOR) was 32.56 months, median progression-free survival (mPFS) was 30.13 months.

Cilta-cel is part of a global strategic collaboration between Legend and Janssen, under which the companies jointly share the development, production and commercialisation activities. The deal includes a 50/50 profit-loss sharing agreement, with the exception of Greater China where the agreement is 70/30.

Diana Spencer, Senior Digital Content Editor, DDW