Adynovate (recombinant factor VIII) meets primary endpoint in phase III trial for haemophilia- Baxalta

Baxalta has announced initial results from a Phase III clinical trial of Adynovate (recombinant factor VIII), an extended circulating half-life recombinant Factor VIII (rFVIII) treatment for hemophilia A based on ADVATE [Antihaemophilic Factor (Recombinant)]. The study enrolled 73 previously-treated patients (PTPs) with severe haemophilia A younger than 12 years of age and assessed the treatment's haemostatic efficacy in prophylaxis and treatment of bleeding episodes. All participants received prophylactic Adynovate treatment (median 1.9 infusions per week) and were followed for six months.

Adynovate met its primary endpoint in the study, as no patients developed inhibitory antibodies to Adynovate. In addition, no treatment-related serious adverse events were reported. More than 70 percent (72.7 percent) of patients had no joint bleeds while on treatment with Adynovate and nearly 40 percent (37.9 percent) experienced zero bleeds. The median annualized bleeding rate (ABR) among patient participants treated with Adynovate was 2.0 (range 0-49.8; mean ABR 3.0), which was comparable to the rates seen in the adult study.

Comment: Adynovate was FDA approved in November 2015. Competition comes from a group of of long-acting factor XIII treatments, led by Biogen Idec/Sobi with Eloctate, which can be dosed every three to five days. Novo Nordisk (N8 GP) and Bayer (BAY 94 9027) are nearing approval for similar drugs, which will expand a haemophilia market analysts say could reach more than $11 Bn by 2016.