Axicabtagene gets orphan drug status in JapanDecember 13, 2018
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.