Breaking News! Legend Biotech’s CAR-T product “Ciltacabtagene Autoleucel” has been approved in China

Today (2024-08-27), the National Medical Products Administration (NMPA) of China has granted approval for the CAR-T product “Ciltacabtagene Autoleucel,” developed by Legend Biotech. This new drug application was officially accepted and has now been approved based on priority review by the Center for Drug Evaluation (CDE). The approved indication is for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have previously received at least one proteasome inhibitor and one immunomodulatory agent.

Ciltacabtagene Autoleucel, targeting the B-cell maturation antigen (BCMA), is a chimeric antigen receptor T cell (CAR-T) therapy. It has been recognized globally, with approvals in the United States, European Union, Japan, and other regions. BCMA is a protein that is highly expressed on myeloma cells, making it an ideal target for treatment.

The product, known as “Carvykti” in English, features a distinct structure with a 4-1BB co-stimulatory domain and dual antibody domains aimed at BCMA. In 2017, Legend Biotech entered into a global licensing and collaboration agreement with Janssen Innovation Medicine, part of Johnson & Johnson, to co-develop and commercialize Ciltacabtagene Autoleucel.

The Chinese approval comes after the drug’s new drug application was accepted by the CDE in December 2022 and granted priority review status. The application was supported by the results of the CARTIFAN-1 clinical trial, a pivotal study conducted in China that demonstrated an overall response rate (ORR) of 87.5% with 79.2% of patients achieving a stringent complete response (sCR) after a median follow-up of 26.4 months.

Legend Biotech has also reported long-term follow-up data from the LEGEND-2 study, showing a median overall survival of 55.8 months and an 18% disease-free rate among R/R MM patients after more than five years. Furthermore, the phase 2 CARTITUDE-2 study’s cohort D results indicate that patients who did not achieve complete remission after frontline treatment can attain deep and lasting remissions with Ciltacabtagene Autoleucel, with an ORR of 94%.

Multiple myeloma is a malignant blood cancer with significant unmet clinical needs. Despite advancements in innovative therapies, many patients face disease recurrence and resistance to existing treatments. The approval of Ciltacabtagene Autoleucel in China is expected to bring benefits to numerous patients with this condition, offering a new therapeutic option.