OPKO Health, Inc. has received orphan drug designation from the US Food and Drug Administration (FDA) for its longer-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. These patients are currently being treated by commercially-available Factor VIIa, with estimated 2013 worldwide sales of $1.7 billion.

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 preventive prophylactic treatment of the disease, especially children.

Preclinical data presented by OPKO at the 7th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) on February 26-28, 2014 in Brussels show that OPKO's long-acting Factor VIIa-CTP 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 could potentially change the Factor VIIa market segment, allowing children and adults with hemophilia to easily self-administer the drug at home on a prophylactic basis.

Dr Phillip Frost, CEO of OPKO, commented, "We are pleased with the new orphan drug designation for Factor VIIa-CTP in the US, as it would allow OPKO seven years of marketing exclusivity upon commercialization of the drug. We have previously presented data in animal models of hemophilia showing that Factor Vlla-CTP demonstrates superiority across key efficacy and safety parameters compared to current Factor VIIa therapy. We are enthused about the potential for SC administration due to its improved bioavailability compared to presently available Factor VIIa IV therapy. Expecting to initiate a phase IIa 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 longer acting Factor VIIa product."

FDA's Orphan Drug Act of 1983 was designed to encourage the development of products that demonstrate promise for the diagnosis, prevention and/or treatment of life-threatening or very serious conditions that are rare and affect 200,000 persons or less in the US. The orphan drug designation provides for seven years of market exclusivity, reduction in regulatory fees, certain tax credits and additional regulatory support for R&D initiatives.

OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development and commercialization expertise and novel and proprietary technologies.