Tagraxofusp-erzs (Elzonris™), a CD123-directed cytotoxin, is a fusion protein composed of a recombinant human interleukin-3 and truncated diphtheria toxin administered by intravenous infusion. Tagraxofusp-erzs (Elzonris™) inhibits protein synthesis and causes cell death in CD123- expressing cells. On December 21, 2018, tagraxofusp-erzs (Elzonris™) was approved by the US Food and Drug Administration (FDA) as an orphan drug (a drug used to treat, prevent, or diagnose a rare disease) for the treatment of BPDCN in individuals two years and older.
The safety and efficacy of tagraxofusp-erzs (Elzonris™) was evaluated in a multi-stage, multicenter, open-label, single-arm Phase 2 clinical trial. The trial consisted of 13 treatment-naive individuals and 15 refractory or relapsed individuals with BPDCN. The primary endpoint was clinical complete response. In the treatment-naive cohort, the clinical complete response was 53.8% (7/13) with a complete response duration range of 3.9 to 12.2 months. Median duration of complete response was not reached. In the relapsed or refractory cohort, the clinical complete response of the was 13.3% (2/15).
C86.4 Blastic NK-cell lymphoma