CARVYKTI: half of earlier-line myeloma patients free at five years
Legend Biotech has presented five-year follow-up data from the CARTITUDE-2 Cohort A study of CARVYKTI (ciltacabtagene autoleucel; cilta-cel) in patients with relapsed or refractory multiple myeloma (RRMM) who had received one to three prior lines of therapy. Ten of the 20 patients in the cohort (50%) remained alive and progression-free at five years after a single infusion, without maintenance therapy. Median progression-free survival was 60.5 months, and median overall survival had not been reached at the time of analysis, with 69.2% of patients alive at the five-year mark.
The data were presented at the 2026 International Myeloma Society Annual Meeting in Glasgow and build on previously reported long-term outcomes from CARTITUDE-1, which evaluated cilta-cel in a later-line setting. Legend Biotech and its collaborator Johnson & Johnson position the findings as evidence that earlier deployment of the therapy may improve the probability of durable, treatment-free remission.
The data in detail
The cohort carried a median follow-up of 60.7 months. The safety profile at five years was described as consistent with the established profile for cilta-cel, with no new CAR T-cell-related neurotoxicity observed. Since the previous analysis at approximately 30 months, one patient developed acute myeloid leukaemia and two deaths occurred, attributed to progressive disease and a new cancer respectively. The boxed warning for CARVYKTI includes risk of secondary haematological malignancies, cytokine release syndrome, and neurological toxicities, all of which remain considerations for treating clinicians.
Niels van de Donk, Professor of Haematology at Amsterdam UMC and a consultant to Legend Biotech, said he believed the findings, "showing half of the patients in the cohort remain progression- and treatment-free at five years, are unique in multiple myeloma and give us reason for optimism and deepen our understanding of what may be possible when CARVYKTI is used earlier in the treatment journey."
Market and competitive context
The multiple myeloma CAR-T space has become one of the most commercially significant segments in cell therapy. Bristol Myers Squibb's idecabtagene vicleucel (ide-cel; Abecma) is the other approved BCMA-directed CAR-T, though cilta-cel has generally reported stronger efficacy outcomes in comparative trial data, and the two products occupy different label positions. The broader competitive landscape also includes bispecific antibodies targeting BCMA and GPRC5D, which offer an off-the-shelf alternative without the manufacturing lead time associated with autologous cell therapies.
The CARTITUDE-2 cohort is small at 20 patients, which limits the statistical weight of the findings; nonetheless, five-year durability data in any myeloma treatment setting remain rare and are likely to feature in ongoing label-expansion discussions. Cilta-cel received US FDA approval for second-line RRMM in April 2024, broadening access beyond the heavily pre-treated population in which it was first approved in 2022. The EMA granted conditional marketing authorisation in May 2022 and Japan's MHLW approved the therapy in September of that year. Future milestones will include longer follow-up from the larger, randomised CARTITUDE-4 study, which has shown a numerically higher early mortality rate in the treatment arm, a finding that regulators and prescribers will continue to monitor as real-world use in earlier lines expands.