MIAMI--(BUSINESS WIRE)--OPKO Health, Inc. (NYSE:OPK) today 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 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. Factor VIIa-CTP already obtained orphan status in the U.S. 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 hemophilia to be easily self-administered at home on a prophylactic basis.
Dr. Phillip Frost, CEO 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 commercialization of the drug. We have previously presented data in animal models of hemophilia 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 hemophilia 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.”
This press release contains "forward-looking statements," as that term is defined under the Private Securities Litigation Reform Act of 1995 (PSLRA), regarding product development efforts and other non-historical facts about our expectations, beliefs or intentions regarding our business, technologies and products, financial condition, strategies or prospects, including statements regarding our expectations about our 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, whether Factor VIIa-CTP has the potential for substantial improvement of the quality of life of patients, via both IV and subcutaneous (SC) administration, whether Factor VIIa-CTP will be able to be administered subcutaneously using a simple injection and allow children and adults with hemophilia to easily self-administer the drug at home on a prophylactic basis, whether we will be able to initiate a Phase IIa trial with our IV formulation in hemophilia patients in late 2014, whether we will be in a position to be first to market with a longer acting Factor VIIa product, expectations about market potential for Factor VIIa-CTP, whether we will be able to successfully develop, obtain approval for and launch sales of the Factor VIIa-CTP, and the expected completion dates for our trials. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements. These factors include those described in our filings with the Securities and Exchange Commission, as well as risks inherent in funding, developing and obtaining regulatory approvals of new, commercially-viable and competitive products and treatments, including the risks that clinical trials for Factor VIIa-CTP may not be successful or achieve the expected results or effectiveness, and may not generate data that would support the approval or marketing of this product for the indications being studied, that others may develop products which are superior to Factor VIIa-CTP, and that Factor VIIa-CTP may not have advantages or prove to be superior over presently marketed products. In addition, forward-looking statements may also be adversely affected by general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new products and indications, manufacturing issues that may arise, patent positions and litigation, among other factors. The forward-looking statements contained in this press release speak only as of the date the statements were made and we do not undertake any obligation to update forward-looking statements. We intend that all forward-looking statements be subject to the safe-harbor provisions of the PSLRA.