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European approval for cilta-cel as second-line R/R multiple myeloma treatment

A type II variation of ciltacabtagene autoleucel (cilta-cel; brand name Carvykti) has been approved by the European Commission (EC) for use in the treatment of eligible patients with multiple myeloma, its manufacturer Janssen has announced.

Cilta-cel has been recommended for adult patients with relapsed and refractory (R/R) multiple myeloma, who have received at least one prior therapy including an immunomodulatory agent and a proteasome inhibitor, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide.

Cilta-cel is a chimeric antigen receptor (CAR) T-cell therapy directed against B-cell maturation antigen (BCMA) and is the first BCMA CAR-T therapy approved in Europe for the treatment of eligible patients as early as first relapse.

The approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use in March 2024.

Professor Jesús San Miguel, director of clinical & translational medicine at the University of Navarra in Pamplona, Spain, said: ‘Patients with lenalidomide-refractory multiple myeloma tend to experience early resistance to standard treatments and their disease worsens exponentially with each additional line of therapy.

‘A single infusion of cilta-cel has been shown to significantly lower the risk of progression or death compared to current treatment options, as early as after first relapse.’

Cilta-cel infusion and progression-free survival

The EC approval was based on the results of a phase 3 CARTITUDE-4 clinical trial evaluating the efficacy and safety of cilta-cel in patients with relapsed and lenalidomide-refractory multiple myeloma.

Data from the study were previously published in the New England Journal of Medicine.

The primary endpoint was progression-free survival (PFS), defined as the time from randomisation to the first documentation of disease progression or death.

In total, 419 patients with relapsed and lenalidomide-refractory multiple myeloma who had received one to three prior lines of therapy underwent randomisation.

Patients were randomised to receive either a sequence of apheresis, bridging therapy, lymphodepletion and cilta-cel (n=208) or standard of care, which included daratumumab, pomalidomide and dexamethasone (DPd) or pomalidomide, bortezomib and dexamethasone (PVd) (n=211).

At a median follow-up of 15.9 months, a single infusion of cilta-cel gave rise to a significantly lower risk of disease progression or death compared to standard care (hazard ratio, HR = 0.26, 95% CI 0.18 – 0.38, p < 0.001).

The median duration of PFS was not reached in the cilta-cel arm and was 11.8 months in the standard care arm (95% CI, 10-14).

After 12 months, PFS was 75.9% (95% CI 69.4 – 81.1) in the cilta-cel group and 48.6% (95% CI 41.5 – 55.3) in the standard care group. In addition, a higher proportion of patients in the cilta-cel group had an overall response (84.6% vs. 67.3%), a complete response or better (73.1% vs 21.8%) and an absence of minimal residual disease (60.6% vs. 15.6%).

In terms of safety, Grade 3 or 4 adverse events occurred in a similar proportion of participants in the two groups (96.6% vs 94.2%, cilta-cel vs standard care).