EU orphan designations for OPKO's clotting Factor VIIa-CTP

The European Committee for Orphan Medicinal Products (COMP) gave a positive opinion recommending the approval of US drugmaker OPKO Health’s (NYSE: OPK) 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 hemophilia 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, the company noted. Factor VIIa-CTP already obtained  orphan status in the USA earlier this year. The product was acquired last year through  OPKO’s $480 million purchase of PROLOR.

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.