FDA Approves Treatments for Two Rare Blood Disorders

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.