
Cilta-Cel's Efficacy in Heavily Pretreated Patients With Multiple Myeloma: Thomas G. Martin, MD
Therapy Status
Carvykti (ciltacabtagene autoleucel)
Johnson & Johnson / Legend Biotech
Approved for relapsed/refractory multiple myeloma after at least 1 prior line of therapy (lenalidomide-refractory). Boxed warnings cover CRS, ICANS, Parkinsonism/GBS, HLH/MAS, prolonged cytopenias, immune effector cell-associated enterocolitis (added October 2025), and secondary hematologic malignancies. The CAR-T REMS program was eliminated in June 2025.
- February 28, 2022 · FDA approval for r/r multiple myeloma after ≥4 prior lines
- April 19, 2024 · Class-wide boxed warning added: secondary T-cell malignancy
- June 26, 2025 · FDA eliminates REMS for approved CAR-T therapies
- October 10, 2025 · Boxed warning added: immune effector cell-associated enterocolitis Latest
The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed data from the CARTITUDE-1 study presented at ASH 2021.
“In my mind, these [data from CARTITUDE-1] were the best results that have ever been shown in this truly triple-class refractory, 6 prior lines of therapy population. This is the most aggressive and the most responsive and efficacious therapy, as a single agent in refractory multiple myeloma treatment, that we've ever seen.”
Ciltacabtagene autoleucel (cilta-cel) produced deep and durable responses in heavily-pretreated patients with multiple myeloma, according to data from the phase 1/2 CARTITUDE-1 study (NCT03548207) presented at the
Investigators found that the 97 participants treated with cilta-celin the CARTITUDE-1 study had an overall response rate of 98% and that of 61 evaluable participants with minimal residual disease, the progression-free survival rate at 2 years was 100%. Also presented at ASH 2021 were positive results in a less-pretreated population.3
GeneTherapyLive spoke with Martin to learn more about the data from CARTITUDE-1 presented at ASH 2021. He discussed the positive results and cilta-cel's potential in this population.




















