Factor VIIa Treatment Application for Investigational New Drug Submitted

Hemophilia ReportsJune 2015
Volume 2
Issue 2

Treatment for hemophilia A and B patients with inhibitors to factor VIII or factor IX has been submitted as an Investigational New Drug to the FDA.

A phase 2a study of a long acting coagulation Factor VIIa has been submitted to the US Food and Drug Administration (FDA), according to a company press release.

OPKO Health, Inc., a multinational biopharmaceutical and diagnostics company based in Miami, Florida, announced that they submitted an Investigational New Drug Application (IND) to the FDA in order to investigate the drug’s efficacy and safety for hemophilia A or B patients with inhibitors to factor VIII or factor IX.

The long acting coagulation factor VIIa (factor VIIa CTP) is a novel recombinant factor VIIa which utilizes OPKO’s proprietary technology in order to extend its circulatory half life without the use of polymers, encapsulation techniques, or nano particles, according to the company press release. The statement further explained that the technology is based on a naturally occurring peptide called the C-terminal peptide (CTP), and comes from the beta chain of human chorionic gonadotropin.

Factor VIIa is only available right now as an intravenous formula which requires multiple infusions into the patient to treat a bleeding episode due to the factor VIIa’s short half life. The statement noted that these frequent infusions are particularly inconvenient when being used as preventative prophylactic treatment therapy, and especially in the case of children with hemophilia.

“We look forward to beginning clinical trials of Factor VIIa CTP,” Phillip Frost, MD, Chairman and CEO of OPKO Health, said in the statement. “A longer-acting Factor VII administered either intravenously or by subcutaneous administration could change the Factor VIIa market by permitting children and adults to easily self administer at home on a prophylactic basis.”

Orphan drug designation has been granted to Factor VIIa CTP in the United States and Europe. So far, pre clinical studies of intravenous and subcutaneous formulation of factor VIIa in animal models of hemophilia A and B has demonstrated its duration of action and significantly increased survival in the models.

OPKO also uses CTP in their hGH CTP, a long acting recombinant human growth hormone product the company produces. The hGH CTP is currently being evaluated in a phase 3 clinical trial for adults and a phase 2 clinical trial for children with growth hormone deficiencies. OPKO previously announced its collaboration with Pfizer to develop and commercialize hGH CTP for global use.

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