Janssen seeks EU approval for first CAR-T therapy cilta-cel

Johnson & Johnson’s (J&J) pharma division Janssen has filed its first CAR-T cell therapy ciltacabtagene autoleucel (cilta-cel) with the European Medicines Agency (EMA), seeking approval for the treatment of patients with relapsed/refractory multiple myeloma.

The marketing authorisation application (MAA) is supported by results from Janssen’s ongoing Phase Ib/II CARTITUDE-1 study, which is investigating the safety and efficacy of cilta-cel.

Results from this study were recently presented at the virtual American Society of Hematology (ASH) annual meeting, showing ‘very high’ overall response rates (ORR).

In the results, 97% of patients achieved a response, with 67% achieving a stringent complete response (sCR) at a median follow-up of 12.4 months.

Meanwhile, median progression-free survival (PFS) was not reached at the median follow-up, with the 12-month PFS rate found to be 77% and a 12-month overall survival rate of 89%.

The trial included 97 patients treated with cilta-cel who received a median of six prior lines of therapy, with 88% being triple-refractory and 99% refractory to the last line of therapy.

“Despite advances in the treatment of multiple myeloma, there remains a high unmet need, especially for patients whose disease continues to progress,” said Peter Lebowitz, global therapeutic area head, oncology, Janssen research & development.

“Through our collaboration with Legend Biotech, we continue to expedite the development of cilta-cel with a focus and priority on the patients who may benefit from this novel immunotherapy in the future,” he added.

In 2017, Janssen entered into a license and collaboration agreement with Legend Biotech to develop and commercialise cilta-cel, which was followed by the initiation of the CARTITUDE-1 trial in May 2018.

The EMA previously granted cilta-cel a Priority Medicines (PRIME) designation in 2019, while the European Commission granted the CAR-T therapy an orphan designation in 2020.