Bone Marrow Failure Disorders
FDA Approves New Treatment for Adults With Serious Rare Blood Disease
On May 18, 2021, the FDA announced its approval of Empaveli (pegcetacoplan) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Empaveli, manufactured for Apellis Pharmaceuticals, Inc., is the first PNH treatment to target complement protein C3.
U.S. Food and Drug Administration
