The U.S. Food and Drug Administration (FDA) approved an expanded indication for Adynovate® (antihemophilic factor [recombinant], PEGylated) for pediatric patients (<12 years) with hemophilia A. The drug also was approved for use in surgical settings for both pediatric and adult patients.
The decision for pediatric approval was based on data from a prospective, uncontrolled, open-label, multicenter, phase III study that demonstrated that antihemophilic factor (recombinant), PEGylated did not cause previously treated pediatric patients to develop inhibitory antibodies (the study’s primary endpoint). Most patients (73%) had no joint bleeds (n=48/66), and 38 percent did not experience bleeds (n=25/66). The median overall annualized bleeding rate (ABR) was 2.0 (mean = 3.04; range = 2.21-4.19 ABR), which was similar to the rates seen in studies of an adult population. No serious adverse events (AEs) were reported.
The approval to allow the treatment in surgical settings was based on interim results of an ongoing phase III study of perioperative control of hemostasis among 15 patients with severe hemophilia A undergoing surgical procedures. The most common AEs associated with antihemophilic factor (recombinant), PEGylated include headache and nausea.
Source: Shire Pharmaceuticals press release, December 27, 2016.