Elzonris Receives Approval to Treat Rare Blood Cancer

December 21, 2018 – The U.S. FDA has approved ElzonrisTM (tagraxofusp-erzs), manufactured by Stemline Therapeutics, to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN) in patients two years of age and older. Elzonris is the first FDA-approved therapy for BPDCN.

A rare and aggressive cancer that affects the bone marrow and blood cells, BPDCN is often misdiagnosed as non-Hodgkin lymphoma (NHL), acute myeloid leukemia (AML), leukemia cutis, melanoma, or lupus erythematosus. The average age of diagnosis is 60 to 70 years old, with men diagnosed more frequently than women. Little data currently exists on the most effective first-line treatment options, and the standard of care prior to the FDA’s approval of Elzonris has been intensive chemotherapy followed by bone marrow transplants.

In one clinical study, 54% of patients treated with Elzonris in the first cohort trial achieved complete remission or complete remission with a skin abnormality not indicative of active disease. In a second cohort, only two out of 15 patients achieved the same result.

Recommended dosing with Elzonris is 12mcg/kg administered via intravenous (IV) infusion over the course of 15 minutes once daily on the first five days of a 21-day cycle. The first cycle must be administered in an inpatient setting. Subsequent cycles can be conducted in either an inpatient or outpatient setting, as appropriate. Prior to each treatment, patients should be pre-medicated with an H1-histamine antagonist, acetaminophen, a corticosteroid, and an H2-histamine antagonist. A black box warning cautions that patients treated with Elzonris may be at increased risk of life-threatening or fatal capillary leak syndrome.

Elzonris will be available at a wholesale acquisition cost (WAC) of $24,430 per 1mg vial. Stemline Therapeutics is expected to launch Elzonris in early 2019.