Long term study of Eloctate confirms safety and efficacy in haemophilia A.- Orphan Biovitrum.

Orphan Biovitrum announced the final results of ASPIRE the most comprehensive long-term studies of extended half-life factor therapies in haemophilia A. The data confirm the established safety and sustained efficacy of Elocta (efmoroctocog alfa), marketed as Eloctate in the United States and other countries over four years of treatment in previously treated adult, adolescent, and paediatric patients with severe haemophilia A.

ASPIRE is an open-label, non-randomised, multi-year extension study for people who completed the pivotal, phase III A-LONG or Kids A-LONG studies. The primary endpoint is the development of inhibitors. Key findings include the fact that overall median annualised bleed rates (ABRs) for those on prophylactic treatment remained low throughout ASPIRE, particularly in the individualised dosing arm. Zero spontaneous joint bleeds were reported in subjects in all age groups in the individualised dosing arm. Median joint ABRs of below 0.66 were also reported in the cohort.

In the study, adult and adolescent subjects (n=72) treated prophylactically with efmoroctocog alfa experienced a mean improvement in modified hemophilia joint health (mHJHS) score of -2.5 (negative shows improvement) compared to baseline score in A-LONG. Over 92 per cent of subjects either lengthened or experienced no change in dosing intervals during the length of the study. Low ABRs as well as improved joint health scores reported in ASPIRE demonstrate clinical benefit of efmoroctocog alfa that goes beyond just bleed prevention. These results were presented at the 60th Annual Meeting of the American Society of Hematology (ASH).

Comment: Elocta was approved in the EU on 25 November 2015 and by the FDA as Eloctate on 6 June 2014.