Biogen Announces Longer-Acting FVIII Study Results in Kids
Biogen Idec and Swedish Orphan Biovitrum AB have released results from “Kids A-LONG,” a phase 3 clinical study that evaluated the safety, efficacy and pharmacokinetics (measurement of the presence of a drug in the body over time) of ELOCTATE™ in previously treated children under 12 years old with severe hemophilia A.
ELOCTATE™ is the companies’ investigational recombinant factor VIII Fc fusion protein (rFVIIIFc) developed for the treatment of individuals with severe hemophilia A. The technology is designed to keep the infused clotting factor circulating in the body longer, prolonging its therapeutic effects.
According to a Biogen press release, when participants in Kids A-LONG were treated with ELOCTATE™ the therapy was generally well tolerated, no inhibitors were detected and low spontaneous annualized bleeding rates (ABR) were reported. Measurements of efficacy showed that twice-weekly prophylactic dosing with ELOCTATE™ maintained low bleeding rates in children.
Another positive result was that no inhibitors developed during the study. The relative increase in half-life in children with severe hemophilia A was consistent with the 1.5-fold increase in half-life seen in the A-LONG study of adults and adolescents. Children treated prophylactically with ELOCTATE™ had an overall median ABR of 2.0 and a median ABR for spontaneous bleeds of 0.0. Further, 46% of participants in the study experienced no bleeds. In addition, 93% of bleeds were controlled by 1-2 infusions of ELOCTATE™.
“Prophylactic treatment is recommended for children with severe hemophilia due to its proven health benefits. However, current prophylactic infusion schedules are challenging for many children with hemophilia A and their parents,” said Glenn Pierce, MD, PhD, senior vice president of Hematology, Cell and Gene Therapies at Biogen Idec. “The results of this study support the potential for ELOCTATE to address a significant need for children with hemophilia A by providing prolonged intervals between scheduled prophylactic infusions to protect against bleeding episodes.”
Source: Biogen press release dated April 10, 2014