The U.S. Food and Drug Administration (FDA) has approved Stemline Therapeutics, Inc.’s 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.

BPDCN is an aggressive, orphan hematologic malignancy with historically poor outcomes and is an area of unmet medical need. It may present with features similar to, and can be mistaken for, certain diseases, including acute myeloid leukemia, non-Hodgkin’s lymphoma, acute lymphocytic leukemia, myelodysplastic syndromes, and chronic myelomonocytic leukemia, as well as other malignancies with skin manifestations.

The malignancy typically presents in the bone marrow and/or skin, and may also involve lymph nodes and viscera. The diagnosis is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56.

CD123 is a key marker in identifying BPDCN and is a rapidly emerging target for therapeutic research in a variety of cancers. Elzonris is designed to specifically target CD123, and, within a triad of signature markers, enables proper diagnosis.

Elzonris was granted Breakthrough Therapy Designation and Orphan Drug Designation, and the Elzonris Biologics License Application was evaluated under Priority Review by the FDA. 

Stemline intends to bring Elzonris to patients globally. In November 2018, the European Medicines Agency granted accelerated assessment to the upcoming Elzonris Marketing Authorization Application submission, which is expected in the first quarter of 2019.

(Source: Stemline Therapeutics, Inc.)