FDA Approves Tagraxofusp, First Approved Treatment for a Rare, Aggressive Hematologic Cancer

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US Food and Drug Administration
US Food and Drug Administration

Tagraxofusp package insert includes a boxed warning alerting physicians and patients of an increased risk of capillary leak syndrome, a condition in which fluid and proteins leak out of the tiny blood vessels into surrounding tissues. The condition can be life-threatening or fatal to patients in treatment.

Common side effects reported in the clinical trials included nausea, fatigue, peripheral edema, fever, chills, and weight increase. Most common laboratory abnormalities reported included decreases in lymphocytes, albumin, platelets, hemoglobin, and calcium, and increases in glucose, ALT, and AST. Healthcare providers are advised to monitor patients' liver enzyme levels and for signs of intolerance to the infusion. Use in pregnant or breastfeeding women is not recommended as tagraxofusp may harm a developing fetus or newborn baby.

US FDA granted the application Breakthrough Therapy Designation and Orphan Drug Designation; the Biologics License Application was evaluated under priority review. The European Medicines Agency (EMA) granted accelerated assessment to the tagraxofusp Marketing Authorization Application (MAA) submission, which is expected in early 2019.

Other indications for tagraxofusp being investigated include chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and others. 


1. FDA approves first treatment for rare blood disease [news release]. US Food & Drug Administration website; December 21, 2019. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm629020.htm. Accessed January 7, 2019.

2. FDA approves Elzonris™ (tagraxofusp), the first treatment for blastic plasmacytoid dendritic cell neoplasm and first CD123-targeted therapy [news release]. New York, NY: Stemline Therapeutics Inc. https://www.prnewswire.com/news-releases/fda-approves-elzonris-tagraxofusp-the-first-treatment-for-blastic-plasmacytoid-dendritic-cell-neoplasm-and-first-cd123-targeted-therapy-300770317.html?tc=eml_cleartime. Access January 7, 2019.
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