Nippon Shinyaku : ELZONRIS® I.V. Injection Receives Marketing Approval for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm in Japan

Kyoto, Japan,December 22 , 2025 - Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto; President: Toru Nakai) today announced that the Ministry of Health, Labour and Welfare (MHLW) has approved ELZONRIS® I.V. Injection (Tagraxofusp (genetically recombinant)) for the treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) in Japan. Nippon Shinyaku acquired a license for ELZONRIS® I.V. Injection (Tagraxofusp (genetically recombinant)) for the treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) in Japan. Nippon Shinyaku acquired a license for ELZONRIS® from Menarini Group (Headquarters: Florence, CEO: Elcin Barker Ergun) in March 2021, and has been advancing its development in Japan.

BPDCN is a rare aggressive hematologic malignancy that has features of both leukemia and lymphoma, with characteristic skin lesions, lymph node involvement, and frequent spread to the bone marrow, with poor prognosis. In the absence of an approved treatment option, BPDCN treatment is based on intensive multiagent leukemia or lymphoma chemotherapy regimens in Japan. However, the median overall survival of patients with BPDCN continues to be short (less than 12 months) and new therapeutic agents are desired^1-2.

ELZONRIS® is a first-in-class CD123 targeted therapy that induces apoptosis of BPDCN cells by inhibiting protein synthesis by specifically targeting CD123 expressing BPDCN cells. Based on the results of Phase I/II trials conducted overseas in untreated and relapsed/refractory BPDCN patients, ELZONRIS® has been approved by the U.S. Food and Drug Administration for the treatment of BPDCN in adults and in paediatric patients 2 years and older in December 2018, and by the European Medicines Agency for adult patients with untreated BPDCN in January 2021. ELZONRIS® has already been approved in over 40 countries worldwide.

The marketing authorization is based on data from the domestic Phase I/II study in Japanese BPDCN patients as well as overseas Phase I/II trials conducted by Stemline Therapeutics Inc, of the Menarini Group.

Nippon Shinyaku will work to ensure appropriate access to ELZONRIS® for patients in need and expects that ELZONRIS® will contribute to the treatment of BPDCN.

We believe that by delivering ELZONRIS® to patients in need, we can contribute to the treatment of BPDCN in Japan.

Summary of Approval