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Home » FDA Approves Treatments for Two Rare Blood Disorders
FDA Approves Treatments for Two Rare Blood Disorders
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January 2, 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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