The U.S. FDA approved antihemophilic factor (recombinant), PEGylated for use in patients 12 years old or older with hemophilia A. The new drug is approved for on-demand treatment and control of bleeding episodes, as well as to reduce the frequency of bleeding episodes. The drug is a modified version of recombinant factor VIII, with an extended circulating half-life to last longer in the blood and potentially require less frequent injections than unmodified antihemophilic factor when used to reduce the frequency of bleeding.
The approval of antihemophilic factor (recombinant), PEGylated was based on the results of a prospective, multicenter, open-label, non-randomized, phase III clinical trial of 137 adults and adolescents with hemophilia A. Patients were assigned to either twice-weekly prophylaxis (40-50 IU/kg; n=120) or on-demand treatment (10-60 IU/kg; n=17) with the modified factor.
Previously treated patients in the twice-weekly prophylaxis arm had 95 percent fewer annual bleeds compared with those treated on-demand (median annual bleed rate = 1.9 vs. 41.5, respectively). During the study, 38 percent of prophylaxis-treated patients experienced zero bleeds. Moreover, 57 percent of patients experienced zero joint bleeds based on six months of prophylaxis. No safety concerns were identified during the trial.
Source: U.S. Food and Drug Administration press release, November 13, 2015.