OPKO Health, Inc., recently announced the submission of an Investigational New Drug Application (IND) to the US Food and Drug Administration (FDA) to conduct a Phase 2a study of a long-acting version of its recombinant factor VIIa (Factor VIIa-CTP). The therapy is intended for the treatment of bleeding episodes in people with hemophilia A or B with inhibitors to factor VIII or factor IX. OPKO is a multinational biopharmaceutical and diagnostics company based in Miami, Florida.
Factor VIIa-CTP is being developed through OPKO’s proprietary technology, which extends the therapies circulatory half-life using a naturally occurring peptide (biological molecules made up of amino acid chains) known as the C-terminal peptide (CTP). CTP is also being used by OPKO in the manufacturing of a long-acting recombinant human growth hormone product currently being evaluated for treatment in children with growth hormone deficiencies.
According to an OPKO press release, pre-clinical studies of both intravenous and subcutaneous versions of Factor VIIa-CTP “demonstrated its duration of action and significantly increased survival,” in animal models.
“We look forward to beginning clinical trials of Factor VIIa-CTP,” said Phillip Frost, MD, Chairman and CEO of OPKO Health. “A longer acting Factor VIIa administered either by IV or subcutaneous administration could change the factor VIIa market by permitting children and adults to easily self-administer at home on a prophylactic basis.”
Source: OPKO press release dated January 30, 2015
