Emerging therapies for haemophilia - Global perspective

The therapeutic options for people with haemophilia (PWH) have rapidly evolved in the last 5 years. Moving on from conventional plasma-derived and recombinant clotting factor concentrates (CFC), there now are extended half-life CFCs (~1.8× for FVIII and ~4.5× for FIX) to as well as several novel haemostasis agents administered subcutaneously (weekly to monthly) such as bispecific antibody which brings together FIXa with FX like FVIII, a liver-targeted siRNA against antithrombin which can reduce its levels enough to allow significant haemostasis and an antibody against tissue factor pathway inhibitor which then also enhances haemostasis. Successful gene therapy for both haemophilia A and haemophilia B has been demonstrated by gene transfer using adeno-associated virus vectors. Sustained clinically significant elevation (>5%) to normal factor levels has been demonstrated. Some of these products have already obtained market authorization whilst others are at various stages of development. The choices of products for the treatment of haemophilia have never been better. Whilst the immediate superiority of all these products providing better haemostasis and convenience than conventional CFCs, their exact position in the clinical algorithm will need to be defined based on the long-term safety and efficacy data. However, most of these products are likely to remain out of reach of >70% of PWH in the world. The biggest challenge will be to find and establish mechanisms for wider access to these transformational haemostasis products for all PWH around the world.