EMA Recommends Gene Therapy for Treatment of Multiple Myeloma
INNOVATIVE personalised gene therapy for the treatment of multiple myeloma (MM) has been recommended by the European Medical Association (EMA) to be granted conditional marketing authorisation.
Ciltacabtagene autoleucel (cilta-cel) is a chimeric antigen receptor (CAR) T cell therapy, sold under the brand name Carvykti™, which is used for the treatment of relapsed and/or refractory MM in patients who have not responded to immunomodulatory agents, proteasome inhibitors, and monoclonal antibodies. In patients where first-line treatments have been unsuccessful, cilta-cel can be an effective and personalised therapeutic alternative, which works by collecting and modifying a person’s own T cells, that are then infused back into the patient.
The EMA recommendation was issued based on the findings of the ground-breaking CARTITUDE trial. This study was designed to investigate the efficacy and safety profile of cilta-cel, and involved 97 patients who had already undergone prior treatment for MM, receiving one dose of cilta-cel. The results showed that 78% of patients who responded to treatment presented a stringent complete response, where symptoms or signs of disease were undetectable. The overall survival rate was 81%, according to the latest results available.
The CARTITUDE study remains ongoing, as the safety profile of cilta-cel needs to be continuously assessed to evaluate its long-term effects in light of some concerns regarding common side effects of the treatment, which include neurological toxicity, cytopenia, serious infections, and in particular, cytokine release syndrome.
Commenting on the findings from the study, principal investigator Sundar Jagannath, Director of the Center of Excellence for Multiple Myeloma, and Professor of Medicine, Haematology, and Medical Oncology at the Tisch Cancer Institute of the Icahn School of Medicine at Mount Sinai, New York, USA, said: “The responses in the CARTITUDE-1 study showed durability over time and resulted in the majority of heavily pretreated patients achieving deep responses after 18-month follow-up.” He added: “The approval of cilta-cel provides physicians an immunotherapy treatment option that offers patients an opportunity to be free from anti-myeloma therapies for a period of time.”