Ciltacabtagene autoleucel receives FDA approval for R/R multiple myeloma

The U.S. Food and Drug Administration (FDA) has approved the use of ciltacabtagene autoleucel (cilta-cel), a BCMA-directed chimeric antigen receptor T-cell (CAR-T) immunotherapy, for the treatment of patients with relapsed/refractory (R/R) multiple myeloma who have received four or more prior lines of therapy, including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and an anti-CD38 antibody.1

Despite recent advances in treatment regimens, multiple myeloma remains a largely incurable disease with a poor survival outcome.2 Therefore, novel immunotherapies such as cilta-cel are important for patients in the relapsed/refractory setting.

The recent approval of cilta-cel is based on the results of the CARTITUDE-1 trial (NCT03548207). At 18-month follow-up, 98% of patients treated with cilta-cel achieved an overall response rate (ORR), with 80% of patients achieving stringent complete response (sCR).1,3 At median follow-up of 22 months, the percentage of patients who achieved sCR increased to 83%, with cilta-cel demonstrating a consistent safety profile and no new safety signals. In addition, of the 61 patients evaluable for minimal residual disease (MRD), 92% achieved MRD negativity at 10-5. 3

Dr Niels van de Donk, MD, PhD, of the VU University Medical Center, Amsterdam, Netherlands, discussed some results from the CARTITUDE-1 and CARTITUDE-2 (NCT04133636) studies at the American Society of Hematology meeting in 2021. Dr van de Donk emphasizes the deep and durable responses observed in patients, saying, “I think cilta-cel is a good drug for people with aggressive early relapse, and these patients, again, have a high risk of early mortality because of the aggressiveness of the disease”:

The growing field of CAR-T therapy is changing the treatment landscape for R/R myeloma treatment, with cilta-cel being the second CAR-T product to be approved for myeloma following the approval of idecabtagene vicleucel (ide-cel) in 2021.4 These novel immunotherapies are very promising, providing more hope for patients with R/R myeloma, and excitement for clinicians in this area. 


  1. Johnson & Johnson. U.S. FDA Approves CARVYKTI™ (ciltacabtagene autoleucel), Janssen’s First Cell Therapy, a BCMA-Directed CAR-T Immunotherapy for the Treatment of Patients with Relapsed or Refractory Multiple Myeloma. Available from: (Last accessed 02/03/22).
  2. Bird SA, Boyd K. Multiple myeloma: an overview of managementPalliat Care Soc Pract. October 2019; 13; DOI: 1178224219868235.
  3. Johnson & Johnson. New Data from CARTITUDE-1 Study Show Continued Deep and Durable Responses of Ciltacabtagene Autoleucel (cilta-cel) in Treatment of Heavily Pretreated Patients with Multiple Myeloma. Available from: (Last accessed 02/03/22).
  4. US Food and Drug Administration. FDA approves idecabtagene vicleucel for multiple myeloma. Available from: (Last accessed 02/03/22).

Written by Anya Dragojlovic Kerkache

Edited by Thomas Southgate