US orphan designation for OPKO's longer-acting clotting Factor VIIa-CTP

The US Food and Drug Administration has granted orphan drug designation to US drugmaker OPKO Health’s (NYSE: OPK) longer-acting version of clotting Factor VIIa (Factor VIIa-CTP) for the treatment of bleeding episodes in patients with hemophilia 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, the company noted. 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 Hemophilia 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.