Adynovate (recombinant factor VIII) approved by FDA for hemophilia A, in pediatric patients under 12 years of age and in surgical settings- Shire

Shire announced that the FDA has approved Adynovate (recombinant factor VIII) for hemophilia A, in pediatric patients under 12 years of age. The FDA also approved Adynovate for use in surgical settings for both adult and pediatric patients.

The approval of Adynovate to treat children under the age of 12 was based on data from a prospective, uncontrolled, open-label, multi-center Phase 3 trial designed to assess the immunogenicity along with the safety and efficacy of Adynovate. Results from the study showed that Adynovate met its primary endpoint with no previously treated children having developed inhibitory antibodies to Adynovate. In addition, no treatment-related serious adverse events were reported. More than 70 percent (73 percent) of children had zero joint bleeds (n=48/66) while on prophylactic treatment with Adynovate and nearly 40 percent (38 percent) experienced zero bleeds (n= 25/66). The median overall annualized bleeding rate (ABR) among pediatric patients treated with Adynovate was 2.0 (mean ABR 3.04; range 2.21–4.19), which was similar to the rates seen in the adult study. Approval to use Adynovate in surgical settings for both adult and pediatric patients 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.

Comment: Adynovate was first approved by the FDA in November 2015.