An alternative to C5 inhibitors, Apellis’ pegcetacoplan wins FDA approval in PNH May 17, 2021 By Lee Landenberger Now that Apellis Pharmaceuticals Inc.’s Empaveli pegcetacoplan has won FDA approval as the first targeted C3 therapy for treating paroxysmal nocturnal hemoglobinuria (PNH), the cost of treating the rare blood disorder is hefty.Read More
Fulcrum Therapeutics' FTX-6058 could be a disease-modifying treatment for sickle cell disease April 26, 2021