Data from FDA Approval Summary: Tagraxofusp-erzs For Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm

• DOI: 10.1158/1078-0432.c.6529523.v1
• Publication Date: 2023-04-01T06:19:48Z
• Authors: Emily Y. Jen; Xin Gao; Liang Li; Luning Zhuang; Natalie E. Simpson; Baikuntha Aryal; Rong Wang; Donna Przepiorka; Yuan Li Shen; Ruby Leong; Chao Liu; Christopher M. Sheth; Steven Bowen; Kirsten B. Goldberg; Ann T. Farrell; Gideon M. Blumenthal; Richard Pazdur
• Keywords: Peripheral edema

<div>Abstract<p>Tagraxofusp-erzs (Elzonris, Stemline) is a cytotoxin that targets CD123-expressing cells. On December 21, 2018, FDA approved tagraxofusp-erzs for the treatment of blastic plasmacytoid dendritic cell neoplasms (BPDCN) in adult and pediatric patients 2 years older. Approval was based on response rate single-arm trial, Study STML-401-0114; pivotal cohort included 13 with treatment-naïve BPDCN who received monotherapy. The complete or clinical (CR/CRc) 54% (95% CI: 25%–81%), median duration CR/CRc not reached follow-up 11.5 months (range: 0.2–12.7). In separate exploratory cohort, achieved by (13%) R/R BPDCN. Safety assessed 94 myeloid treated at dose schedule. major toxicity capillary leak syndrome (CLS), which occurred 55% fatal 2%. Hepatotoxicity hypersensitivity reactions were reported 88% 46% patients, respectively. Other common (≥30%) adverse nausea, fatigue, peripheral edema, pyrexia, weight increase. A high proportion (85%) developed neutralizing antidrug antibodies. Tagraxofusp-erzs first FDA-approved BPDCN.</p></div>