TOKYO and BASKING RIDGE, N.J., October 3, 2018 /PRNewswire/ — The Japan Ministry of Health, Labour and Welfare (MHLW) granted the Orphan Drug designation to axicabtagene ciloleucel (KTE-C19, Daiichi Sankyo) for the treatment of diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL), high-grade B-cell lymphoma (HGBL), and transformed follicular lymphoma (TFL), which are all aggressive forms of non-Hodgkin lymphoma (NHL).

Axicabtagene ciloleucel is a chimeric antigen receptor T-cell (CAR-T) therapy directed against CD19. The drug enables the patient’s innate immune system to eradicate certain types of B-cell lymphoma cells. Axicabtagene ciloleucel was previously approved in the United States (US) and Europe based on the results of the Phase 1/2 ZUMA-1 study. A similar design study to the ZUMA-1 is currently being planned in Japan.

Daiichi Sankyo obtained the exclusive development, manufacturing and commercialisation rights in Japan for axicabtagene ciloleucel from Kite Pharma, a Gilead company. In the US and Europe, the CAR-T therapy is marketed as Yescarta®.

The Orphan Drug designation system by the Japan MHLW is designed to promote research and support the development of therapies for severe and difficult-to-treat diseases affecting less than 50,000 Japanese patients, and for which a major unmet medical need.

References

Daiichi Sankyo. (2018, October 03) Japan MHLW Grants Orphan Drug Designation to Axicabtagene Ciloleucel for Treatment of Certain Types of B-Cell Lymphoma [Press Release]. Retrieved from https://www.daiichisankyo.com/media_investors/media_relations/press_releases/detail/006911.html