Legend Biotech’s ciltacabtagene autoleucel (cilta-cel), marketed as Carvykti in the United States and Europe, has been approved by China’s NMPA for treating adults with relapsed or refractory multiple myeloma (r/r MM) who have received three or more lines of therapy, including at least one proteasome inhibitor and one immunomodulatory agent.
“The approval of cilta-cel in the Chinese market is a significant milestone and will provide substantial benefits to many patients,” said Ying Huang, PhD, CEO of Legend Biotech. “We remain committed to our goal of curing patients, expanding our clinical research, and improving access to this innovative therapy for more individuals.”
The NMPA’s decision was based on results from the phase 2 CARTIFAN-1 clinical trial (NCT03758417) conducted at multiple sites in China. Among 58 patients evaluated for efficacy, the overall response rate was 87.9%, with 79.3% achieving a complete response (CR) or stringent CR. Additionally, 86.2% reached a very good partial response or better. The median duration of response was 32.56 months, median progression-free survival was 30.13 months, and the median overall survival was not reached.
In the U.S., cilta-cel is approved for adults with relapsed and lenalidomide-refractory MM who have undergone at least one prior line of therapy, which must include a proteasome inhibitor and an immunomodulatory agent. This expanded indication was approved by the FDA in April 2024, following its original approval in February 2022 for patients who had received four or more prior therapies, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. The April 2024 decision was informed by the CARTITUDE-4 clinical trial (NCT04181827), a phase 3, randomized, open-label study comparing cilta-cel to standard of care in patients who had received one to three prior lines of therapy.
“CARVYKTI has shown remarkable efficacy as a personalized, one-time infusion for the earlier treatment of relapsed/refractory multiple myeloma, as demonstrated in the CARTITUDE-4 study,” stated Binod Dhakal, MD, Associate Professor at the Medical College of Wisconsin, Division of Hematology and Oncology. “With this approval, I am excited for patients who may have the chance for a treatment-free period after their first relapse, alleviating the burden of continuous treatment for this challenging disease.”
Later in April 2024, the European Commission (EC) followed the FDA’s lead, approving cilta-cel for an expanded indication in adults with r/r MM who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and who have shown disease progression on their last therapy while being lenalidomide-refractory. This decision was also based on data from CARTITUDE-4. Cilta-cel had previously received conditional marketing authorization from the EC in May 2022 for adults with r/r MM who had undergone at least three prior therapies, which included a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody, with disease progression occurring during the last treatment.
