FDA approves tagraxofusp-erzs for blastic plasmacytoid dendritic cell neoplasm
- December 27, 2018
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On December 21, 2018, the Food and Drug Administration approved tagraxofusp-erzs(ELZONRIS™, Stemline Therapeutics), a CD123-directed cytotoxin, for blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients 2 years and older.
Approval was based on a multicenter, multicohort, open-label, single-arm clinical trial (STML-401-0114; NCT 02113982) in patients with untreated or relapsed/refractory BPDCN. Patients received tagraxofusp-erzs at the recommended dose of 12 mcg/kg (see schedule below) In the pivotal cohort, seven (53.8%; 95% CI: 25.1, 80.8) of 13 patients with untreated BPDCN achieved complete response/clinical complete response after a median follow-up of 11.5 months. The median response duration was not reached. In the second cohort, of 15 patients with relapsed or refractory BPDCN, one patient achieved a complete response (duration 111 days) and one patient achieved a clinical complete response (duration 424 days).
Most 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.
The recommended tagraxofusp-erzs dose and schedule is 12 mcg/kg administered intravenously over 15 minutes once daily on days 1 to 5 of a 21-day cycle.
FDA granted this application priority review and breakthrough therapy designation. This therapy also received orphan drug designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.
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