Posted by Michael Wonder on 11 Feb 2019
Apellis Pharmaceuticals’ APL-2 receives fast track designation from the FDA for the treatment of patients with paroxysmal nocturnal haemoglobinuria
11 February 2019 - New designation now includes all PNH patients.
Apellis Pharmaceuticals today announced that the U.S. FDA has granted fast track designation to the Company’s APL-2, a novel inhibitor of complement factor C3, for the treatment of patients with paroxysmal nocturnal haemoglobinuria (PNH). APL-2 is currently being studied in a Phase 3 active comparator trial in patients with PNH who remain anaemic despite treatment with eculizumab.
This new fast track designation for APL-2 for all patients with PNH, supersedes the prior fast track designation granted on 15 December 2016 for APL-2 for the subset of PNH patients who continue to experience haemolysis requiring blood transfusions despite receiving therapy with eculizumab.
