Biopharmaceutical firm CSL Behring has started the Phase II / III clinical trial, which has been designed to assess the recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP or CSL689) to treat patients with congenital haemophilia A or B with inhibitors.
The study has been designed to assess the pharmacokinetics (PK), efficacy, and safety of the VIIa with albumin (rVIIa-FP) for on-demand treatment in patients with congenital haemophilia A or B who have developed an inhibitor to factor VIII or factor IX replacement therapy.
The company will enrol around 54 male patients in the study, of which first was enrolled in Malaysia.
CSL R&D director and chief scientific officer Dr Andrew Cuthbertson said: "CSL Behring has a thorough understanding of the bleeding disorders community, focused scientific expertise and a strong commitment to developing and delivering innovative specialty biotherapies that treat serious medical conditions.
"Our commitment, expertise and understanding helped CSL to develop rVIIa-FP, based on the innovative recombinant albumin fusion technology platform, to treat patients with haemophilia A or B with inhibitors as well as congenital factor VII deficiency."
According to the firm, preclinical studies showed that rVIIa-FP has favourable pharmacokinetic properties compared with the existing recombinant FVIIa product.
In the Phase I study in healthy volunteers, the rVIIa-FP demonstrated a good tolerance and a three to four-fold increase in half-life compared with the commercially available rFVIIa-product.
The company received orphan drug status from the European Commission for rVIIa-FP to treat patients with haemophilia A or B who have developed an inhibitor as well as the treatment of congenital factor VII deficiency.
It also obtained orphan drug status from the US Food and Drug Administration for the treatment and prophylaxis of bleeding episodes in patients with congenital haemophilia and inhibitors to coagulation factor VIII or IX.