• Emily Y Jen, Xin Gao, Liang Li, Luning Zhuang, Natalie E Simpson, Baikuntha Aryal, Rong Wang, Donna Przepiorka, ShenYuan LiYLCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Ruby Leong, Chao Liu, Christopher M Sheth, Steven Bowen, Kirsten B Goldberg, Ann T Farrell, Gideon M Blumenthal, and Richard Pazdur.
• Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland. emily.jen@fda.hhs.gov.
• Clin Cancer Res. 2020 Feb 1; 26 (3): 532-536.

AbstractTagraxofusp-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 and older. Approval was based on the response rate in a single-arm trial, Study STML-401-0114; the pivotal cohort included 13 patients with treatment-naïve BPDCN who received tagraxofusp-erzs monotherapy. The complete response or clinical complete response (CR/CRc) rate in the pivotal cohort was 54% (95% CI: 25%-81%), and the median duration of CR/CRc was not reached with a median follow-up of 11.5 months (range: 0.2-12.7). In a separate exploratory cohort, a CR/CRc was achieved by 2 (13%) patients with R/R BPDCN. Safety was assessed in 94 patients with myeloid neoplasms treated with tagraxofusp-erzs at the approved dose and schedule. The major toxicity was capillary leak syndrome (CLS), which occurred in 55% of patients and was fatal in 2%. Hepatotoxicity and hypersensitivity reactions were reported in 88% and 46% of patients, respectively. Other common (≥30%) adverse reactions were nausea, fatigue, peripheral edema, pyrexia, and weight increase. A high proportion of patients (85%) developed neutralizing antidrug antibodies. Tagraxofusp-erzs is the first FDA-approved treatment for BPDCN.©2019 American Association for Cancer Research.

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