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FDAnews Drug Daily Bulletin

FDA Approves Treatments for Two Rare Blood Disorders

Jan. 3, 2019

The FDA approved two candidates for treatment of rare blood disorders in children and adults.

The agency approved Stemline Therapeutics’ Elzonris (tagraxofusp-erzs) infusion for treatment of blastic plasmacytoid dendritic cell neoplasm. The FDA based its approval on an efficacy study in which seven of 13 patients achieved complete remission or remission with a skin abnormality.

The agency also approved Alexion’s Ultomiris (ravulizumab) injection, a long-acting complement inhibitor that prevents hemolysis, to treat paroxysmal nocturnal hemoglobinuria (PNH). A clinical trial involving 246 patients previously untreated for PNH found the drug noninferior to eculizumab.

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