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news.Biogen Idec and Swedish Orphan Biovitrum (Sobi) have reported positive top-line results of the Kids B-LONG Phase III clinical trial of Alprolix [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in children under age 12 with severe hemophilia B.
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The trial, which evaluated the safety, efficacy and pharmacokinetics of Alprolix, met its primary endpoint and achieved low bleeding rates with once-weekly prophylactic dosing in children.
Alprolix is the only approved recombinant, clotting factor IX therapy with prolonged circulation in the body.
During the trial, Alprolix was generally well tolerated and no inhibitors were detected, while once-weekly prophylactic dosing with Alprolix resulted in low bleeding rates.
The Phase III Kids B-LONG trial examined the safety, efficacy and pharmacokinetics of Alprolix in previously treated children under age 12 with severe hemophilia B.
Primary endpoint of the trial was to assess the occurrence of inhibitor development, while secondary endpoints included the overall and spontaneous annualized bleeding rates (ABR).
ABR is the estimated number of yearly bleeding episodes and the number of infusions used to treat bleeding episodes.
Biogen Idec vice president of hematology clinical development Aoife Brennan said: "According to published studies, prophylactic treatment for children with severe hemophilia is recommended because it is associated with proven clinical benefits.
"However, frequent administration schedules can be burdensome for children and their caregivers.
"These data will enable regulatory filings in Europe later this year as well as support pediatric indications in other countries, with the potential to help address a critical need among children with hemophilia B."
The successful completion of the trial supports applications for pediatric indications in several geographies and is a major step in seeking marketing authorization for Alprolix in Europe.
The European Medicines Agency (EMA) requires the inclusion of pediatric trial data in the initial marketing application for a new hemophilia therapy.
The company said that interim results from the Phase III Kids B-LONG trial helped support the US approval of Alprolix for use in children.