Axicabtagene gets orphan drug status in Japan

December 13, 2018 0 By FM

Axicabtagene ciloleucel has been granted Orphan Drug designation by the Japan Ministry of Health, Labour and Welfare (MHLW) 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 aggressive forms of non-Hodgkin lymphoma (NHL).
Axicabtagene ciloleucel is a chimeric antigen receptor T cell (CAR T) therapy directed against CD19, which harnesses a patient’s own immune system to fight certain types of B-cell lymphoma.
Based on the results of a phase 1/2 study (ZUMA-1), axicabtagene ciloleucelhas been approved in the US and Europe. A phase 2 study similar to the ZUMA-1 study is currently being planned in Japan.
The Japan MHLW Orphan Drug designation system is designed to promote research activities and support the development of orphan drugs for serious, difficult-to-treat diseases that affect fewer than 50,000 patients in Japan, and for which significant unmet medical need exists.