EC expands use of BCMA-targeted CAR-T therapy

CAR-T therapy

The European Commission (EC) has approved a Type II variation for Carvykti (ciltacabtagene autoleucel; cilta-cel) for the treatment of adult patients with relapsed and refractory multiple myeloma (RRMM).

It is approved in patients who have received at least one prior therapy, including an immunomodulatory agent (IMiD) and a proteasome inhibitor (PI), who  have demonstrated disease progression on the last therapy, and are refractory to lenalidomide.

Cilta-cel is a chimeric antigen receptor T-cell (CAR-T) therapy directed against B-cell maturation antigen (BCMA), a protein that is highly expressed on myeloma cells.

More than 35,000 new cases of multiple myeloma were diagnosed in the European Union in 2022.

Edmond Chan, EMEA Therapeutic Area Lead Haematology, Johnson & Johnson Innovative Medicine, said: “Today’s approval marks important progress for eligible patients with multiple myeloma, who may now benefit from treatment with cilta-cel earlier in their treatment pathway, where it has the potential to transform outcomes and change the trajectory of their disease.”

Results of the CARTITUDE-4 study

The expanded indication for cilta-cel is based on positive results from the Phase III CARTITUDE-4 study, evaluating the efficacy and safety of cilta-cel in patients with relapsed and lenalidomide-refractory multiple myeloma.

At a median follow-up of 15.9 months, a single infusion of cilta-cel resulted in a significantly lower risk of disease progression or death versus SOC. The median duration of progression-free survival (PFS) was not reached in the cilta-cel arm and was 11.8 months in the SOC arm. At 12 months, estimated PFS rate was 76% in the cilta-cel arm and 49% in the SOC arm.

Patients in the cilta-cel arm achieved an 85% overall response rate (ORR) and 73% achieved a complete response (CR) or better. Among patients in the SOC arm, the ORR was 67% and CR or better was 22%. Overall minimal residual disease (MRD) negativity rate was higher in the cilta-cel arm (61%) than the SOC arm (16%).

“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,” said Professor Jesús San Miguel, Director of Clinical & Translational Medicine, Universidad de Navarra, Spain. “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.”

The EC also approved the conversion of the conditional marketing authorisation (MA) for cilta-cel to a standard MA, as the obligations of the conditional approval have now been met.

The US Food and Drug Administration (FDA) approved cilta-cel for the same indication on 5 April 2024.

Diana Spencer, Senior Digital Content Editor, DDW

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