OPKO Health, Inc, announced that the European Committee for Orphan Medicinal Products (COMP) gave a positive opinion recommending the approval of orphan drug designation for OPKO's long-acting version of clotting Factor VIIa (Factor VIIa-CTP) for the treatment of bleeding episodes in patients with haemophilia A or B with inhibitors to Factor VIII or Factor IX and congenital Factor VII deficiency. These patients are currently being treated by commercially-available Factor VIIa, with estimated 2013 worldwide sales of $1.7 billion. Factor VIIa-CTP already obtained orphan status in the US earlier this year.

Currently, Factor VIIa therapy is available only as an intravenous (IV) formulation, which requires multiple injections to treat a bleeding episode due to Factor VIIa's short half-life. In addition, the requirement for multiple weekly injections can be onerous for patients interested in preventative prophylactic treatment of the disease, especially children.

Preclinical data previously presented by OPKO show that OPKO's long-acting Factor VIIa-CTP has demonstrated the potential for substantial improvement of the quality of life of patients, via both IV and subcutaneous (SC) administration. The combination of a long-acting Factor VIIa coupled with SC administration using a simple injection would potentially change the Factor VIIa market segment, allowing children and adults with haemophilia to be easily self-administered at home on a prophylactic basis.

Dr. Phillip Frost, chief executive officer, of OPKO, commented, "We are pleased that the COMP will recommend orphan designation for Factor VIIa-CTP in the EU, as it would allow OPKO 10 years of marketing exclusivity upon commercialisation of the drug. We have previously presented data in animal models of haemophilia showing that Factor Vlla-CTP demonstrates superiority of key efficacy and safety parameters compared to current Factor VIIa products. We are excited about the potential for SC administration. This may represent another major competitive advantage for our long-acting Factor Vlla-CTP. We expect to initiate a Phase IIa clinical trial with our IV formulation in haemophilia patients in late 2014. We believe that we are in a unique position to be first to market with a long acting Factor VII-a therapy."