The U.S. Food and Drug Administration (FDA) approved BAY94-9027, or antihemophilic factor (recombinant), PEGylated-aucl, for the prophylactic treatment of adults and adolescents (≥12 years) with previously treated hemophilia A. The FDA also approved the therapy for on-demand treatment and the perioperative management of bleeding in the same population.
The recombinant factor VIII replacement therapy was approved based on results from the phase II/III PROTECT VIII trial of previously treated adults and adolescents, which demonstrated bleed protection and safety to a median of 1.9 years (range = 0-2.6 years).
The initial recommended prophylactic regimen for BAY94-9027 is twice weekly, with the ability to dose every five days and individually adjust dosing based on bleeding episodes. The most frequently reported adverse events were headache, cough, nausea, and fever.
Sources: FDA approval letter, August 29, 2018; Bayer press release, August 30, 2018.