Extension Study Data Supporting Long-Term Safety and Efficacy of Eloctate Published in Haemophilia

Biogen and Swedish Orphan Biovitrum AB (publ) (Sobi) have announced newly published clinical data demonstrate that people on extended-interval prophylaxis regimens with Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein] experienced low bleeding rates. The interim results of this Phase III, open-label extension study called ASPIRE were published in the online edition of Haemophilia, the journal of the World Federation of Hemophilia, the European Association for Haemophilia and Allied Disorders, and the Hemostasis & Thrombosis Research Society.

Study participants completing the Phase III A-LONG and Kids A-LONG studies were eligible to participate in ASPIRE. The results to-date show the majority of participants in ASPIRE, maintained or extended their dosing intervals between treatments compared to the A-LONG and Kids A-LONG studies. As of the interim analysis, the median time in the ASPIRE study was 80.9 weeks for adults and adolescents completing the A-LONG study, and 23.9 weeks for children completing the Kids A-LONG study. Inhibitor development is the primary endpoint of ASPIRE and no inhibitors were reported in any treatment groups. Through the interim ASPIRE analysis, adults and adolescents experienced annualized bleeding rates (ABRs) of 0.66, 2.03 and 1.97 in the individualized, weekly and modified prophylaxis arms, respectively. Children on individualized prophylaxis also experienced low bleeding rates, with an overall median ABR of 0.0 in children less than 6 years of age, and 1.54 for children ages 6 to 12. These results were consistent with data from the Phase III A-LONG and Kids A-LONG studies.

In addition to efficacy and safety endpoints, the publication also reports changes in prophylactic infusion frequency from the end of the A-LONG study through the interim analysis. Of the adults and adolescents who had previously been treated prophylactically and who remained in the study through the interim analysis (n=128), 72% maintained their prophylactic dosing interval and 22% lengthened and 6% shortened the time between infusions. Extension study participants could change treatment group at any time.

“The design of the ASPIRE study provides physicians a high degree of dosing flexibility, with the goal of reflecting their real-world treatment practices,” said Guy Young, Director of the Hemostasis and Thrombosis Center, Children’s Hospital of Los Angeles. “The results suggest prophylaxis with Eloctate shows efficacy and safety for the long-term treatment of hemophilia A.”

In ASPIRE, most participants received prophylactic treatment and were able to maintain protection against bleeding episodes with Eloctate consumption that was consistent with that observed in A-LONG and Kids A-LONG.