FDA Approves Stemline’s Elzonris for Rare Blood Disease

FDA has approved Stemline Therapeutics’ Elzonris (tagraxofusp-erzs) infusion to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients age two and older. An agency announcement says BPDCN is an aggressive and rare disease of the bone marrow and blood that can affect multiple organs, including the lymph nodes and skin. It often presents as leukemia or evolves into acute leukemia.

Elzonris’ efficacy was studied in two cohorts of patients in a single-arm clinical trial. Common side effects reported by patients in trials were capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia, chills, and weight increase.

The labeling for Elzonris contains a Boxed Warning about an increased risk of capillary leak syndrome that may be life-threatening or fatal to patients in treatment.

The application had been granted breakthrough designation, priority review, and orphan drug designation.