Tagraxofusp Receives Orphan Drug Designation in Japan for BPCDN

Elcin Barker Ergun

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

References

1. The Menarini Group announces Elzonris (tagraxofusp) designated as an orphan drug for BPDCN by Japanese Ministry of Health, Labor and Welfare. News release. Menarini Group. August 30, 2023. Accessed August 31, 2023. https://www.menarini.com/en-us/news/news-detail/the-menarini-group-announces-elzonris174-tagraxofusp-designated-as-an-orphan-drug-for-bpdcn-by-japanese-ministry-of-health-labor-and-welfare
2. FDA approves tagraxofusp-erzs for blastic plasmacytoid dendritic cell neoplasm. News release. FDA. December 21, 2018. Accessed August 31, 2023. https://www.fda.gov/drugs/fda-approves-tagraxofusp-erzs-blastic-plasmacytoid-dendritic-cell-neoplasm
3. Menarini receives European Commission approval of Elzonris (tagraxofusp), for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). News release. Menarini Group. January 21, 2021. Accessed August 31, 2023. https://www.menarini.com/en-us/news/news-detail/menarini-receives-european-commission-approval-of-elzonris-tagraxofusp-for-the-treatment-of-blastic-plasmacytoid-dendritic-cell-neoplasm-bpdcn