Overview of Ciltacabtagene Autoleucel Approval
Overview of Ciltacabtagene Autoleucel Approval
Ciltacabtagene Autoleucel: An Overview of Its Approval Journey
From 2014 to 2022, it took Ciltacabtagene Autoleucel eight years to complete the journey from research and development to clinical trials. Through the continuous research and innovation of the R&D team, the outstanding clinical data has demonstrated that Ciltacabtagene Autoleucel is an effective drug. Next, global commercialization is the necessary path for Ciltacabtagene Autoleucel’s growth. The collaboration with the international pharmaceutical giant Johnson & Johnson in 2017 was a crucial step in achieving its global strategy. Based on the excellent clinical data, in December 2019, the FDA granted Ciltacabtagene Autoleucel “Breakthrough Therapy Designation”; in February 2020, the European Commission granted it “Orphan Drug Designation”; on June 5, 2020, Legend Biotech officially went public on NASDAQ. In August 2020, Ciltacabtagene Autoleucel also received the first “Breakthrough Therapy” certification from the China National Medical Products Administration (NMPA) Center for Drug Evaluation.
Currently, in collaboration with Johnson & Johnson, Ciltacabtagene Autoleucel has been launched in the United States, European Union, and Japan.
Ciltacabtagene Autoleucel Approved in the United States
On February 28, 2022, Legend Biotech officially announced that Ciltacabtagene Autoleucel was approved by the U.S. FDA for the treatment of relapsed or refractory multiple myeloma (R/R MM) patients who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
Ciltacabtagene Autoleucel is the seventh CAR-T product approved globally, the sixth CAR-T product approved by the U.S. FDA, and the first China-originated CAR-T cell therapy product approved in the United States. This approval was primarily based on the results of the pivotal Phase Ib/II CARTITUDE-1 (NCT03548207) study.
The latest data showed that Ciltacabtagene Autoleucel demonstrated a high overall response rate of 98% in patients with relapsed or refractory multiple myeloma who had received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. In the pivotal CARTITUDE-1 (NCT03548207) study, 97 R/R MM patients experienced early, deep, and durable responses, with an overall response rate (ORR) of 98% (95% CI: 92.7-99.7), and 78% of patients achieved a stringent complete response (sCR, 95% CI: 68.8-86.1). At a median follow-up of 18 months, the median duration of response (DOR) was 21.8 months (95% CI, 21.8-not estimable).
Ciltacabtagene Autoleucel Approved in the European Union
On May 27, 2022, the European Commission granted conditional marketing authorization for Ciltacabtagene Autoleucel for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have received at least three prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody, and have had disease progression on the last line of treatment. This approval was primarily based on the results of the pivotal Phase Ib/II CARTITUDE-1 (NCT03548207) study, which showed that CARVYKTI® demonstrated a high overall response rate of 98% in a single treatment for patients with relapsed or refractory multiple myeloma. This approval was based on the key CARTITUDE-1 (NCT03548207) study results, which included patients who had received a median of six prior lines of therapy (range 3-18), including proteasome inhibitors (PIs), immunomodulatory agents (IMiDs), and anti-CD38 antibodies. The results showed that, at a median follow-up of 18 months (range 1.5-30.5), 97 R/R MM patients who received a single treatment with Ciltacabtagene Autoleucel experienced early, deep, and durable responses, with an ORR of 98% (95% CI: 92.7-99.7). Notably, 80% of patients achieved a stringent complete response (sCR), defined as the absence of detectable disease on imaging or other assessments.
Ciltacabtagene Autoleucel Approved in Japan
On December 6, 2021, Johnson & Johnson submitted a new drug application (NDA) for Ciltacabtagene Autoleucel to the Japanese Ministry of Health, Labor, and Welfare. On September 27, 2022, the Japanese Ministry of Health, Labor, and Welfare approved Ciltacabtagene Autoleucel for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM), limited to the following two conditions: patients with no prior history of treatment with chimeric antigen receptor (CAR) T-cell therapy targeting B-cell maturation antigen (BCMA); patients who have received at least three prior lines of therapy, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD), and an anti-CD38 monoclonal antibody, and were refractory to the last line of treatment or had relapsed. This approval was based on the results of the pivotal Phase 1b/2 CARTITUDE-1 (NCT03548207) study, which included 97 patients who had received a median of six prior lines of therapy (range 3-18), including PIs, IMiDs, and anti-CD38 antibodies. The study showed that non-Japanese patients who received a single treatment with Ciltacabtagene Autoleucel experienced deep and durable responses, with an ORR of 96.9% (95% CI, 91.2-99.4 n=97).
Ciltacabtagene Autoleucel in China
In January 2023, the China National Medical Products Administration (NMPA) Center for Drug Evaluation (CDE) included Ciltacabtagene Autoleucel’s marketing application in the priority review and approval procedure, which will accelerate the domestic approval process in China, with an expected completion this year.
After six years, Ciltacabtagene Autoleucel achieved commercialization in 2022, and its sales are likely to increase year-over-year in the next few years. The global clinical development programs for Ciltacabtagene Autoleucel between Legend Biotech and Johnson & Johnson are also progressing steadily. The collaboration between Legend Biotech and Johnson & Johnson on Ciltacabtagene Autoleucel has become a benchmark in the industry, pursuing shared scientific understanding and mutual interests. This win-win model provides a good direction for the development of China’s biopharmaceutical industry.
Content Source：招财小黄鸭