Local time on September 27, 2022, Legendary Creatures (NASDAQ: LEGN) announced that the Ministry of Health, Labour and Welfare (MHLW) of Japan has approved CARVYKTI® (Ciltacabtagene Autoleucel, Cilta-cel) for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM). Only the following two conditions are met:
The patient had no history of chimeric antigen receptor (CAR) -positive T cell infusion targeting B-cell maturation antigen (BCMA).
The patient had received at least three lines of previous therapy, including proteasome inhibitors (PIs), immunomodulators (IMids), and anti-CD38 monoclonal antibodies, and had not responded to the last treatment or had relapsed.
The new drug application was submitted by legendary biological partner Yangsen. In December 2017, Legendary Bio entered into an exclusive worldwide license and collaboration agreement with Yangsen to develop and commercialize Cilta-cel.
CARVYKTI® features two single-domain antibodies targeting B-cell maturation antigen (BCMA), personalized, and one-time delivery.
This approval is based on the results of the pivotal phase 1B /2 CARTITUDE 1 study, which enrolled 97 patients who had received a median of six lines of previous therapy (range 3-18), including PI, IMiD, and anti-CD38 mab. The study showed that a non-Japanese population receiving one-time treatment with cidalquiolenxil had a deep and durable response, with an overall response rate (ORR) of 96.9% (95%CI, 91.2 to 99.4 n=97). Of note, 65 patients achieved a strictly complete response (sCR), with a strictly complete response rate of 67% (95%CI, 56.7-76.2), meaning that no signs or symptoms of disease could be observed on imaging or other tests after treatment *. The efficacy observed in Japanese patients with multiple myeloma is consistent with that observed in non-Japanese populations. At a median follow-up of 18 months, the median duration of response (DOR) was 21.8 months.
The CARTITUDE 1 study assessed the safety of cedarchiolencel in 106 adult patients, including 97 non-Japanese people and 9 Japanese patients. Of 106 patients treated with cedarchiolencel, 105 (99.1%) had adverse reactions. The most common adverse events included cytokine release syndrome (94.3%), cytopenia (79.2%), neutropenia (75.5%), thrombocytopenia (59.4%), anemia (51.9%), neurologic adverse events (39.6%), infection (19.8%) and hypombulinemia (11.3%) ).
In February this year, CARVYKTI® (Sidarchiolensa) received marketing approval from the US Food and Drug Administration (FDA) and conditional marketing Authorization from the European Commission (EC) in May.
