On July 3, 2023, the China NMPA approved FUCASO (Equecabtagene Autoleucel), the world’s first fully-human anti-B cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell therapy. This groundbreaking treatment is designated for patients with relapsed and/or refractory multiple myeloma (R/R MM) who have undergone three or more prior therapies, including at least one proteasome inhibitor and one immunomodulatory agent.
FUCASO is an autologous BCMA CAR-T cell injection utilizing a lentiviral vector to deliver a CAR structure featuring a fully human single-chain variable fragment (scFv), along with CD8a hinge, transmembrane, 4-1BB co-stimulatory, and CD3ζ activation domains. Administered as a one-time treatment at a recommended dose of 1.0 × 10^6 CAR-T cells/kg, FUCASO demonstrates strong efficacy and prolonged persistence in patients, providing new treatment options for those with R/R MM.
The approval is based on data from the pivotal FUMANBA-1 study (CTR20192510, NCT05066646), conducted across multiple sites in China. Updated findings from this study were presented at the 2023 Annual Meeting of the American Society of Clinical Oncology (ASCO), revealing impressive outcomes from 103 R/R MM patients with a median follow-up of 13.8 months.
Key study results include:
- An overall response rate (ORR) of 96% among 101 evaluable patients, with a stringent complete response (sCR) rate of 74.3%.
- A median time to response of just 16 days and a 12-month progression-free survival (PFS) rate of 78.8%.
- 95% of patients achieved minimal residual disease (MRD) negativity, with all patients who reached sCR also achieving MRD negativity.
- Of the 12 patients with prior CAR-T therapy, 9 responded, including 5 who achieved sustained sCR for over 18 months.
- In the 89 patients without prior CAR-T therapy, the ORR was 98.9%, with 78.7% achieving sCR/CR.
- Among 103 safety-evaluable patients, only one experienced grade ≥3 cytokine release syndrome (CRS), and two had grade 1-2 immune effector cell-associated neurotoxicity syndrome (ICANS), with all recovering fully.
FUCASO remained detectable in 50% of patients after 12 months and 40% after 24 months post-infusion, with only 19.4% testing positive for anti-drug antibodies (ADA).
Professors Lugui Qiu and Chunrui Li, principal investigators of the FUCASO trial, emphasized the significant unmet medical need in China for effective multiple myeloma treatments. They highlighted FUCASO’s impressive efficacy, providing clinicians with a powerful new option for managing R/R MM.
