Positive Ph III pediatric data for investigational hemophilia A drug Eloctate

US biotech firm Biogen Idec (Nasdaq: BIIB) and Swedish Orphan Biovitrum (STO: SOBI) today released positive top-line results of the Kids A-LONG Phase III clinical study that evaluated the safety and efficacy of Eloctate, an investigational recombinant factor VIII Fc fusion protein product candidate, in children with severe hemophilia A.

Eloctate was generally well-tolerated and no inhibitors (neutralising antibodies that may interfere with the activity of the therapy) were detected. Efficacy analyses showed twice-weekly prophylactic dosing with Eloctate maintained low bleeding rates in children.

Kids A-LONG is the first study to evaluate a long-lasting, investigational hemophilia therapy in children under 12 years old. Eloctate was developed using a process called Fc fusion and is designed to prolong the circulation of infused clotting factor in the body. The successful completion of this study will support applications for pediatric indications globally and is an important step to obtaining marketing authorization in Europe. The European Medicines Agency requires the inclusion of pediatric study data in a marketing application for a new hemophilia therapy, the companies noted.