Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.US-based OPKO Health has submitted an investigational new drug (IND) application to the US Food and Drug Administration (FDA) seeking approval to conduct a Phase IIa trial of its long-acting version of coagulation Factor VIIa (Factor VIIa-CTP) to treat bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX.c
Subscribe to our email newsletter
Factor VIIa-CTP is a new, long-acting recombinant Factor VIIa using the company’s proprietary technology to extend its circulatory half-life without the use of polymers, encapsulation techniques or nanoparticles.
The technology is based on a naturally occurring peptide, the C-terminal peptide (CTP) of the beta chain of human chorionic gonadotropin.
The CTP technology is also used in the company’s hGH-CTP, its long-acting recombinant human growth hormone product which is being evaluated in Phase III clinical trials for adults and Phase II trials for children with growth hormone deficiencies.
The company has recently entered into a global agreement with Pfizer to develop and commercialize hGH-CTP.
Currently, Factor VIIa therapy is available only as an intravenous (IV) formulation which, due to Factor VIIa’s short half-life, requires multiple infusions to treat a bleeding episode.
Additionally, frequent infusions are onerous when used as preventative prophylactic therapy, especially for children.
OPKO Health chairman and CEO Phillip Frost said: "A longer acting Factor VII 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."
Pre-clinical studies of intravenous and subcutaneous formulations of the product in hemophilic animal models showed its duration of action and significantly increased survival.
Factor VIIa-CTP has been granted orphan drug designation in both the US and Europe.