The Japanese Ministry of Health, Labor and Welfare has granted an orphan drug designation to tagraxofusp-erzs for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm.
The Japanese Ministry of Health, Labor and Welfare (MHLW) has granted an orphan drug designation to tagraxofusp-erzs (Elzonris; NS-401) for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN).1
“The MHLW’s decision to grant orphan drug designation recognizes the potential positive impact tagraxofusp could have on [patients with] BPDCN in Japan, a patient population that currently has limited treatment options,” Elcin Barker Ergun, chief executive officer of the Menarini Group, stated in a news release. “This achievement by our partner, Nippon Shinyaku, advances our commitment to bring transformative new therapeutic options to patients with difficult-to-treat cancers, and to deliver innovative medicines to people around the globe.”
Nippon Shinyaku is currently developing tagraxofusp in Japan and is conducting a phase 1/2 trial evaluating the CD123-targeted agent in Japanese patients with BPDCN.
In December 2018, the FDA approved tagraxofusp for the treatment of adult and pediatric patients at least 2 years of age with BPDCN.2
That approval was supported by data from a multicenter, multi-cohort, single-arm, phase 1/2 trial (NCT02113982), where findings demonstrated that patients in the pivotal cohort (n = 13) experienced a complete response (CR)/clinical CR rate of 53.8% (95% CI, 25.1%-80.8%) at a median follow-up of 11.5 months. The median response duration was not reached in this cohort.
In another cohort of patients with relapsed/refractory BPDCN (n = 15), 1 patient experienced a CR with a duration of 111 days, and 1 patient achieved a clinical CR with a duration of 424 days.
Regarding safety, the most common adverse effects reported in at least 30% of patients included capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia, and increased weight. The most common laboratory abnormalities that occurred in at least 50% of patients consisted of decreased albumin, platelets, hemoglobin, calcium, and sodium, as well as increased glucose, alanine transaminase, and aspartate transaminase.
In January 2021, the European Commission granted a marketing authorization to tagraxofusp monotherapy for the first-line treatment of adult patients with BPDCN.3