Ciltacabtagene autoleucel
Ciltacabtagene autoleucel, sold under the brand name Carvykti, is an anti-cancer medication used to treat multiple myeloma. Ciltacabtagene autoleucel is a BCMA -directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. Each dose is customized using the recipient's own T-cells, which are collected, genetically modified, and infused back into the recipient.
The most common adverse reactions include pyrexia, hypogammaglobulinemia, musculoskeletal pain, fatigue, infections, diarrhea, nausea, encephalopathy, headache, coagulopathy, constipation, and vomiting. Additional common side effects include neutropenia, lymphopenia and leucopenia, anemia, thrombocytopenia, hypotension, pain of the muscles and bones, high level of liver enzymes, upper respiratory tract infection, diarrhea, hypokalemia, hypocalcemia, hypophosphatemia, nausea, headache, cough, tachycardia, encephalopathy, edema, decreased appetite, chills, fever, tiredness, as well as cytokine release syndrome.
Ciltacabtagene autoleucel was approved for medical use in the United States in February 2022, and in the European Union in May 2022.
Medical uses
Ciltacabtagene autoleucel is indicated for the treatment of adults with relapsed or refractory multiple myeloma after one or more prior lines of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide.Adverse effects
In April 2024, the FDA label boxed warning was expanded to include T cell malignancies. In October 2025, the FDA approved a label boxed warning that ciltacabtagene autoleucel can also cause a severe form of enterocolitis, i.e., inflammation of the small and large intestines termed immune effector cell-associated enterocolitis. This warning followed A study showing that 2.2% of patients with multiple myeloma in 11 different treatment centers developed this form of enteritis.History
The safety and efficacy of ciltacabtagene autoleucel were evaluated in CARTITUDE-1, an open label, multicenter clinical trial evaluating ciltacabtagene autoleucel in 97 participants with relapsed or refractory multiple myeloma who received at least three prior lines of therapy which included a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody and who had disease progression on or after the last chemotherapy regimen; 82% had received four or more prior lines of antimyeloma therapy.The U.S. Food and Drug Administration granted the application for ciltacabtagene autoleucel priority review, breakthrough therapy, and orphan drug designations.