The Japanese Ministry of Health, Labor and Welfare (MHLW) granted orphan drug designation to tagraxofusp for the indication of blastic plasmacytoid dendritic cell neoplasm (BPDCN). Tagraxofusp was approved only for patients with BPDCN.
Japan’s MHLW grants orphan drug designation to treatments designed for fewer than 50,000 patients, especially when there is a significant medical necessity. This designation has the potential to reduce the time needed for regulatory approval in Japan by several months, facilitating quicker patient access to these treatments.
Tagraxofusp is a fusion protein that combines interleukin 3 (IL-3) with diphtheria toxin. This fusion protein effectively eliminates cultured pDCs by attaching to their IL-3 receptors, enabling it to enter the cells and subsequently obstructing their protein synthesis.
Tagraxofusp is being manufactured by Nippon Shinyaku Co, Ltd, in Japan, development partners to the Menarini Group and Stemline Therapeutics, Inc. Currently, Nippon Shinyaku is conducting a phase I/II clinical trial.
Source: Business Wire, August 2023