AFSTYLA was tested in the largest hemophilia A pivotal clinical trial program to date, with 258 previously treated patients with severe hemophilia A (226 received prophylaxis).
Phase I/III, open-label, multicenter, crossover study in patients ≥12 years, and a phase III, open-label, multicenter study in patients <12 years.
175 previously treated male patients with severe hemophilia A (<1% endogenous Factor VIII activity) ages 12 to 65 years, including 14 adolescent patients 12 to 18 years. 174 patients received at least one dose of AFSTYLA and 173 (99%) were evaluable for efficacy. A total of 161 patients (92.5%) completed the study.
84 previously treated male children (35 patients 0 to <6 years and 49 patients ≥6 to <12 years) with severe hemophilia A (<1% endogenous Factor VIII activity). Of the 84 enrolled patients, all received at least one dose of AFSTYLA and 83 (99%) were evaluable for efficacy.