FDA approves Elzonris, first treatment for a rare blood disease

US biotech Stemline Therapeutics (Nasdaq: STML) has been granted  US Food and Drug Administration approval of Elzonris (tagraxofusp-erzs; SL-401) for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adult and pediatric patients two years and older, in both treatment-naïve and previously-treated populations.

Elzonris is the first treatment approved for BPDCN and the first approved CD123-targeted therapy, and the FDA decision comes well before the previously expected February 21, 201.

However, the labeling for Elzonris contains a Boxed Warning, the FDA’s strongest, to alert health care professionals and patients about the increased risk of capillary leak syndrome which may be life-threatening or fatal to patients in treatment.