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SL-401 Emerging as First Targeted Therapy for Rare Blood Cancer

Angelica Welch
Published: Thursday, Aug 16, 2018

Naveen Pemmaraju, MD

Naveen Pemmaraju, MD

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy for which there are few treatment options outside of cytotoxic chemotherapy. A new investigational agent, SL-401 (tagraxofusp, Elzonris), may provide an effective targeted therapy for patients with BPDCN, according to Naveen Pemmaraju, MD.

Findings from a pivotal phase II trial presented at the 2018 European Hematology Association Congress showed that SL-401 was active in this patient population and had a manageable safety profile. Overall response rate (ORR) was 90% for patients treated with SL-401 as frontline therapy, reported Pemmaraju.

In August 2018, the FDA granted a priority review designation to a biologics license application (BLA) for SL-401 as a treatment for patients with BPDCN. The agency is scheduled to decide on the BLA by February 21, 2019.

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