FDA Approves Apellis’ Device for Self-Administration of PNH Drug

Rare Daily Staff

The U.S. Food and Drug Administration approved Apellis Pharmaceuticals Empaveli Inector, a compact, single-use, on-body device designed to enhance self-administration of Empaveli for adults with paroxysmal nocturnal hemoglobinuria.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria, and difficulty breathing.

Empaveli is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of PNH in the United States, European Union, and other countries globally. The therapy is also under investigation for several other rare diseases across hematology and nephrology.

The Empaveli Injector is the first high-volume, subcutaneous on-body delivery system which features several advances to streamline self-administration. The compact device offers patients greater mobility when administering Empaveli. A push button starts the injection, and the hidden needle automatically retracts upon dose completion. The Empaveli Injector is developed in collaboration with Enable Injections.