Posted by Michael Wonder on 21 Dec 2016
Apellis announces FDA fast track designation for APL-2 in PNH
20 December 2016 - C3 inhibitor is in development for the treatment of PNH, both in patients not previously treated with eculizumab, and in patients who continue to experience haemolysis and require RBC transfusions despite receiving treatment with eculizumab.
Apellis Pharmaceuticals announced today that the U.S. FDA has granted fast track designation to the development program for APL-2, a complement C3 inhibitor, in the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), who continue to experience haemolysis and require RBC transfusions despite receiving therapy with eculizumab. PNH is a rare, acquired, potentially life-threatening disease characterised by complement-mediated haemolytic aanemia.
