Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study

Abstract

Introduction:The efficacy and safety of recombinant factor VIII Fc fusion protein (rFVIIIFc) as an extended half-life treatment for severe haemophilia A were demonstrated in the Phase 3 A-LONG and Kids A-LONG studies. Eligible subjects who completed A-LONG and Kids A-LONG could enrol in ASPIRE (NCT01454739), an open-label extension study.This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.

Recombinant FVIIIFc Versus BAY 94-9027 for Treatment of Patients with Haemophilia A: Comparative Efficacy Using a Matching Adjusted Indirect Comparison

“…The Fc portion of rFVIIIFc is part of a naturally occurring pathway with no known inherent toxicity. In all clinical trials of rFVIIIFc, treatment was generally well tolerated, adverse events were consistent with those expected in this patient population, and no inhibitors to rFVIIIFc developed in previously treated patients [5,11,13]. Conversely, treatment with PEGylated BAY 94-9027 is associated with concerns around hypersensitivity reactions and lack of efficacy in paediatric patients aged up to 12 years, as a result of anti-PEG immunity [19,20]; BAY 94-9027 is, therefore, not approved for use in this patient population [17,18].…”

2021 clinical trials update: Innovations in hemophilia therapy

“…Assessment of the long‐term safety and efficacy data of recently licensed FVIII and FIX concentrates continues. Sustained efficacy of bleed control and no unexpected safety signals including for inhibitor development in the previously treated patients (PTPs) have been reported to date from post‐marketing studies and surveillance; however, several studies are ongoing, Table 1 36,37 …”

Resolution of the Expert сouncil on сurrent issues in the treatment of patients with hemophilia A with clotting factor VIII with prolonged half-life (INN – Efmoroctocog alfa)

“…Фармакокинетика, безопасность и эффективность препарата эфмороктоког альфа для профилактики кровотечений у взрослых и детей с гемофилией А были оценены в 2 международных исследованиях, с продолжением наблюдения в объединенном исследовании после их завершения [4][5][6].…”

“…Применение препарата у взрослых позволило достичь медианы периода полувыведения 19 ч с его увеличением в 1,5 раза по сравнению со стандартным рекомбинантным фактором VIII. При оценке эффективности у взрослых были отмечены уменьшение медианы годовой частоты всех кровотечений до 1,6, а спонтанных -до 0, купирование эпизодов кровотечений одной инъекцией у 87,3 % и двумя -у 97,8 % пациентов [4,6].…”

“…В обоих исследованиях медиана частоты инъекций составила 2 раза в неделю, а медиана потребления фактора была < 100 МЕ/кг/нед. При длительном наблюдении не зарегистрировано развития ингибиторов на фоне терапии препаратом эфмороктоког альфа у ранее леченных пациентов [4][5][6].…”