Baxalta Incorporated announced that it has submitted supplemental Biologics License Applications (sBLAs) to the U.S. Food and Drug Administration (FDA) seeking approval for the use of ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] to treat children under the age of 12 with hemophilia A and for use in surgical settings.
ADYNOVATE is the only extended circulating half-life recombinant Factor VIII (rFVIII) treatment for hemophilia A based on the demonstrated efficacy of ADVATE [Antihemophilic Factor (Recombinant)]. ADYNOVATE was approved by the FDA in November 2015 for use in adolescent and adult hemophilia A patients (12 years and older) for prophylaxis to reduce the frequency of bleeding episodes and on-demand treatment and control of bleeding.
The submission of ADYNOVATE to treat children under the age of 12 was based on results of a Phase 3 trial designed to assess the efficacy and safety including immunogenicity of ADYNOVATE. Results from the study showed ADYNOVATE met its primary endpoint and no patients developed inhibitory antibodies to ADYNOVATE. In addition, no treatment-related serious adverse events were reported. More than 70 percent (72.7 percent) of patients had no joint bleeds while on treatment with ADYNOVATE (n=66) and nearly 40 percent (37.9 percent) experienced zero bleeds. The median overall annualized bleeding rate (ABR) among patient participants treated with ADYNOVATE was 2.0 (range 0-49.8; mean ABR 3.0), which was comparable to the rates seen in the adult study.
The filing was also supported by the positive results of a Phase 3 study evaluating the efficacy and safety of ADYNOVATE for the perioperative control of hemostasis among 15 patients with severe hemophilia A undergoing surgical procedures, which was reported in December 2015. The study data demonstrated that ADYNOVATE achieved hemostasis control in the perioperative period (from start of the procedure until discharge or day 14) for patients with severe hemophilia A.
