Updates in clinical trial data of extended half-life recombinant factor IX products for the treatment of haemophilia B

Abstract

Whilst the global prevalence of haemophilia B is less than that of haemophilia A, rapid and remarkable innovations have been made in the development of haemophilia B therapies in the last decade. The most recent developments are the evolution of extended half-life haemophilia B replacement therapies which are designed to reduce the treatment burden associated with prophylactic infusion of factor IX (FIX) to prevent bleeding in haemophilia B participants. Clinical development programmes have culminated in the completion of three phase III studies on extended half-life (EHL) recombinant FIX (rFIX) products and subsequent approval and registration of these in many countries around the world. Current data from the three EHL rFIX clinical studies indicate that these products have acceptable safety profiles with no allergic reactions, thromboembolic phenomena or neutralizing antibodies when given to previously treated adolescent and adults for the prevention of bleeds, for the treatment of bleeds and in the perisurgical haemostasis use. Studies in previously untreated paediatric participants are currently ongoing. The EHL rFIX products have the potential impact to reduce the treatment burden associated with prophylactic infusion of replacement FIX, to treat and prevent bleeds in participants with haemophilia B and to improve the participant's health-related quality of life. The impact of EHL rFIX is likely to be modified by current development of other haemophilia B therapy such as antitissue factor pathway inhibitors and haemophilia B gene therapy. In this review, we aim to provide an update on the safety and efficacy data from the three EHL rFIX clinical studies and to consider their roles in the face of novel haemophilia B therapy currently evolving.

Keywords: efficacy; extended half-life; haemophilia B; pharmacokinetics; recombinant FIX; safety.