The US Food and Drug Administration (FDA) recently approved Baxalta's ADYNOVATE, a new longer- lasting recombinant factor VIII (rFVII) product. The new therapy is indicated for the on-demand treatment of bleeding episodes, and for routine prophylaxis in adult and adolescent patients (12 years and older) with hemophilia A. The production of ADYNOVATE includes the use of polyethylene glycol (PEGylated) molecules that keep the rFVIII circulating in the blood for longer periods, potentially leading to less frequent infusions.
Final FDA approval was based on the results of a phase 3 clinical trial that measured the safety and efficacy of ADYNOVATE in 137 adults and adolescents 12 years of age and older. The subjects came from 20 countries, including the US, Australia, Japan and Spain.
Investigators found that ADYNOVATE demonstrated efficacy in treating hemophilia patients using routine prophylaxis and for on-demand bleeding episodes. The results showed that previously treated patients who received the therapy prophylactically twice weekly experienced 95% fewer annual bleeds compared to those who were treated on-demand. 96% of the bleeding episodes that occurred during the study were controlled with one or two infusions. Lastly, no patients developed inhibitors to the treatment and no safety concerns arose during the study. Adverse effects were only seen in 1% of the subjects, the most common of which were headache and nausea.
“The approval of ADYNOVATE provides an important therapeutic option for use in the care of patients with hemophilia A and reduces the frequency of factor VIII infusions needed to avoid bleeding,” said Karen Midthun, MD, director of the FDA's Center for Biologics Evaluation and Research
Source: FDA news release and Baxalta press release, both dated November 13, 2015
