Biogen Idec and Sobi announce positive top-line efficacy and safety results from a phase-3 paediatric study of investigational therapy Eloctate for haemophilia A.
According to the results Eloctate was generally well-tolerated and no inhibitors 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 haemophilia 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 paediatric indications globally and is an important step to obtaining marketing authorisation in Europe. The European Medicines Agency requires the inclusion of paediatric study data in a marketing application for a new haemophilia therapy.
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 haemophilia A. The primary endpoint of the study was to evaluate the frequency of inhibitor (neutralising antibody) development and none were detected. Secondary endpoints included the overall and spontaneous annualised bleeding rates (ABR), or projected number of yearly bleeding episodes, and the number of injections used to prevent and treat bleeding episodes.
In the study, the relative increase in half-life in children with severe haemophilia 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. Forty-six per cent of participants in the study experienced zero bleeding episodes. Overall, ninety three per cent of bleeding episodes were controlled by one to two injections of Eloctate. Additional analyses of the Kids A-LONG study are ongoing, and the companies plan to present detailed results at a future scientific meeting.
“Sobi and Biogen Idec recognise the importance of advancing treatment options for adults and children with haemophilia,” said Birgitte Volck, M.D., Ph.D., senior vice president development and chief medical officer of Sobi. “The successful completion of the Kids A-LONG study in children under 12 years old is an important step to bringing this potential new treatment option to adults and children with haemophilia A and a milestone that will enable regulatory submission in Europe.”