Biogen Idec and Sobi Announce Positive Top-Line Efficacy and Safety Results from Phase III Alprolix Pediatric Study

Biogen Idec and Swedish Orphan Biovitrum AB (Sobi) have announced positive top-line results of the Kids B-LONG Phase III clinical study that evaluated the safety, efficacy and pharmacokinetics of Alprolix [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in children under age 12 with severe hemophilia B. Alprolix was generally well tolerated and no inhibitors (neutralizing antibodies that may interfere with the activity of the therapy) were detected during the study. In this study, once-weekly prophylactic dosing with Alprolix resulted in low bleeding rates. Alprolix is the only approved hemophilia B therapy with prolonged circulation in the body.

The successful completion of Kids B-LONG supports applications for pediatric indications in several geographies and is an important step in seeking marketing authorization for Alprolix in Europe. The European Medicines Agency requires the inclusion of pediatric study data in the initial marketing application for a new hemophilia therapy. Interim results of the Kids B-LONG study helped support the US approval of Alprolix for use in children.

“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,” said Aoife Brennan, vice president of hematology clinical development at Biogen Idec. “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.”

“Sobi and Biogen Idec are committed to advancing treatment options for adults and children with hemophilia,” said Birgitte Volck, senior vice president of development and chief medical officer at Sobi. “The completion of Kids B-LONG marks an important milestone in the Alprolix development program. We are excited to pursue the next stages of preparation for regulatory filing in Europe and to potentially advance treatment options for people with hemophilia B.”