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Elzonris FDA Approval History

Last updated by Judith Stewart, BPharm on Dec 30, 2018.

FDA Approved: Yes (First approved December 21, 2018)
Brand name: Elzonris
Generic name: tagraxofusp-erzs
Dosage form: Injection
Company: Stemline Therapeutics, Inc.
Treatment for: Blastic Plasmacytoid Dendritic Cell Neoplasm

Elzonris (tagraxofusp-erzs) is a CD123-directed cytotoxin for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN).

  • Tagraxofusp-erzs is a fusion protein composed of recombinant human interleukin-3 (IL-3) and truncated diphtheria toxin (DT) that works by inhibiting protein synthesis and causing cell death in the CD123-expressing cells of BPDCN and other hematologic malignancies.
  • The efficacy of Elzonris was established in a multicenter, multicohort, open-label, single-arm clinical trial (STML-401-0114) of 47 patients with BPDCN, including 32 treatment-naïve and 15 previously-treated patients. In the Stage 3 (pivotal) cohort, 13 patients with treatment-naïve BPDCN had a complete response or clinical complete response (CR/CRc) rate of 53.8 percent.
  • Elzonris is administered intravenously over 15 minutes once daily on days 1 to 5 of a 21-day cycle. The first cycle is administered in an inpatient setting, and subsequent cycles may be administered in either an inpatient or appropriate outpatient setting. Patients are premedicated with an H1-histamine antagonist, acetaminophen, corticosteroid and H2-histamine antagonist prior to each Elzonris infusion.
  • The labeling for Elzonris includes a boxed warning for Capillary Leak Syndrome, which may be life-threatening or fatal if not properly managed. Patients are advised to contact their health care professional if they experience new or worsening edema, weight gain, shortness of breath, and/or hypotension after infusion. Patients should weigh themselves daily.
  • Females of reproductive potential should use acceptable contraceptive methods during treatment, and for at least 1 week after the last dose. Lactating women should not breastfeed during treatment, and for at least 1 week after the last dose.
  • Common adverse reactions (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia and weight increase. Most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, and sodium, and increases in glucose, ALT and AST.

Development timeline for Elzonris

Dec 21, 2018Approval FDA Approves Elzonris (tagraxofusp-erzs), the First Treatment for Blastic Plasmacytoid Dendritic Cell Neoplasm and First CD123-Targeted Therapy
Jun 15, 2018Stemline Therapeutics Announces Presentation of Elzonris (tagraxofusp; SL-401) Preclinical Data in Systemic Sclerosis, an Autoimmune Disorder, at EULAR Congress

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.