(NASDAQ: BIIB) and
Swedish Orphan Biovitrum
AB (publ) (Sobi) (STO: SOBI) today released positive top-line results of the Kids A-LONG Phase 3 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 (neutralizing 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.
“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, M.D., Ph.D., 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.”
Kids A-LONG investigated the safety, efficacy and pharmacokinetics (measurement of the presence of the drug in a person’s body over time) of ELOCTATE in previously treated children under 12 years old with severe hemophilia A. The primary endpoint of the study was to evaluate the frequency of inhibitor (neutralizing antibody) development and none were detected. Secondary endpoints included the overall and spontaneous annualized bleeding rates (ABR), or projected number of yearly bleeding episodes, and the number of infusions used to prevent and treat bleeding episodes.