FDA approves Carvykti for patients with multiple myeloma

US FDA approval based on 98 percent response rate in multiple myeloma patients treated once with Carvykti (ciltacabtagene autoleucel, cilta-cel).


The US Food and Drug Administration (FDA) has approved Carvykti (ciltacabtagene autoleucel; cilta-cel) for the treatment of adults with relapsed or refractory multiple myeloma (RRMM) after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

The approval is based on data from the pivotal Cartitude-1 study, which included patients who had received a median of six prior treatment regimens (range, three to 18) and had previously received a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody. 

Carvykti is a chimeric antigen receptor T-cell (CAR-T) therapy featuring two B-cell maturation antigen (BCMA)-targeting single domain antibodies. In the pivotal Cartitude-1 study, one-time treatment with ciltacabtagene autoleucel resulted in deep and durable responses, with 98 percent of patients with RRMM responding to therapy (98 percent overall response rate [ORR] (n=97). Notably, 78 percent of the patients achieving this level of response (n=76) experienced a stringent complete response (sCR), a measure in which a physician is unable to observe any signs or symptoms of disease via imaging or other tests after treatment. At a median of 18 months follow-up, median duration of response (DOR) was 21.8 months.

Carvykti is only available through a restricted programme under a Risk Evaluation and Mitigation Strategy (REMS) called the Carvykti REMS Programme.

“We are committed to harnessing our science, deep disease understanding and capabilities to bring forward cell therapies like Carvykti as we continue to focus on our ultimate goal of delivering a cure for multiple myeloma,” commented Dr Peter Lebowitz, Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC.

Multiple myeloma is an incurable blood cancer that affects plasma cells, found in the bone marrow. Despite the development of additional treatment options in recent years, most people living with multiple myeloma face poor prognoses after experiencing disease progression following treatment with three major therapy classes, which include an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody.

“The responses in the Cartitude-1 study showed durability over time and resulted in the majority of heavily pre-treated patients achieving deep responses after 18-month follow-up,” stated Dr Sundar Jagannath, Director of the Center of Excellence for Multiple Myeloma and Professor of Medicine, Hematology and Medical Oncology at The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai, and principal study investigator. “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.”

As a personalised medicine, Carvykti treatment requires extensive training, preparation, and certification to ensure a positive experience for patients. Through a phased approach, Janssen and Legend Biotech will activate a limited network of certified treatment centres as the company works to scale its production capacity and increase the availability of Carvykti throughout the US in 2022 and beyond.