The Food and Drug Administration (FDA) has approved Elzonris (tagraxofusp-erzs; Stemline Therapeutics) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients aged ≥2 years.

BPDCN is a rare disease of the bone marrow and blood that can impact several organs, including the lymph node and skin. Prior to the approval, the standard of care was intensive chemotherapy followed by bone marrow transplantation. Elzonris is a CD123-directed cytotoxin composed of recombinant human IL-3 and truncated diphtheria toxin (DT) fusion protein that inhibits protein synthesis and causes cell death in CD123-expressing cells.

Elzonris was evaluated in a multicenter, open-label, single-arm clinical trial (Study 0114; NCT02113982) with one cohort (n=13) that included patients with untreated BPDCN, and a second cohort (n=15) that included patients with relapsed or refractory BPDCN. Study patients received Elzonris 12mcg/kg IV once daily on days 1-5 of a 21-day cycle. In the first cohort, 54% (n=7) of patients achieved complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc). In the second cohort, 1 patient achieved CR and 1 achieved CRc. 

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Capillary leak syndrome, nausea, fatigue, peripheral edema, fever, chills, and weight increase were common adverse effects reported. Laboratory abnormalities included decreases in lymphocytes, albumin, platelets, hemoglobin and calcium, and increases in glucose and ALT/AST.

Elzonris carries a Boxed Warning describing the increased risk of capillary leak syndrome, which may be life-threatening or fatal. It will be available as a 1000mcg/mL preservative-free solution in single-dose vials. 

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