FDA Accepts Biologics License Application, Grants Priority Review for Tagraxofusp in Rare Hematologic Malignancy

The U.S. Food and Drug Administration (FDA) has accepted for filing a biologics license application (BLA) for tagraxofusp (Elzonris, formerly SL-401) for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare hematologic malignancy. The FDA also granted Priority Review for the BLA and has set a target action date of February 21, 2019, under the Prescription Drug User Fee Act.

The FDA grants Priority Review to product applications that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. Tagraxofusp has also been granted Breakthrough Therapy designation and Orphan Drug designation by the agency.

About Tagraxofusp and BPDCN

Tagraxofusp is a novel targeted investigational therapy directed to CD123, a cell-surface receptor expressed on a range of malignancies. A pivotal trial of tagraxofusp in patients with BPDCN was successfully completed, and the drug is also being evaluated in clinical trials in additional indications, including chronic myelomonocytic leukemia and myelofibrosis.

Previously known as natural killer cell leukemia/lymphoma, BPDCN is categorized by the World Health Organization in its Classification of Tumors of Haematopoietic and Lymphoid Tissues (4th edition, 2008) as an acute myeloid leukemia (AML). Most often, BPDCN presents with features of both lymphoma and leukemia. There are few available data about BPDCN, and there is no established treatment.

The skin is the most frequently involved site of disease (80% of cases). However, BPDCN usually progresses with bone marrow involvement and a decrease in red blood cell, white blood cell, and platelet counts. The lymph nodes and spleen may also be involved. Rashes without symptoms can also occur. Common misdiagnoses for BPDCN include non-Hodgkin lymphoma, AML, leukemia cutis, melanoma, and lupus erythematosus.

To learn more, visit the Leukemia & Lymphoma Society Web page about BPDCN.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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