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FDA Accepts HEMA Biologics' BLA for Coagulation Factor VIIa Recombinant

RDR Staff
Published Online: Monday, Jan 09, 2017
The US could be in for a potential new treatment for hemophilia A and B patients suffering with inhibitors, as HEMA Biologic’s Biologic License Application (BLA) for Coagulation Factor VIIa, Recombinant (eptacog beta activated) was accepted by the FDA. 
 
HEMA’s BLA submission followed results from their Phase 3 study on the safety, pharmacokinetics, and efficacy of Coagulation Factor VIIa (recombinant) in congenital hemophilia A or B patients with inhibitors to Factor VIII or IX. 
 
In the study, 27 hemophilia patients were administered 2 initial dose regimens in bleeding events. The study met its primary endpoint of hemostatic success. 85% of the patient’s bleeding events treated with the initial 225 µg/kg dose required no further therapy.
 
About HEMA’s Coagulation Factor VIIa, Recombinant Eptacog Beta, Activated
Eptacog Beta activated is a recombinant form of human Factor VIIa. Eptacog beta is currently under clinical development.

About the FDA’s BLA

According to the FDA, The Biologics License Application (BLA) is a request for permission to introduce, or deliver for introduction, a biologic product into interstate commerce.

About hemophilia A

People with hemophilia A, a rare genetic bleeding disorder, do not have or make enough of an essential blood clotting protein called factor VIII. Depending on the severity of the disorder, they can be susceptible to severe, uncontrolled bleeding, especially into the joints.
 
Standard treatment for hemophilia A usually involves intravenous infusions of replacement factor VIII. However, approximately one in four people develop antibodies, or inhibitors, which can bind to and prevent these replacement factors from working. This is a major challenge and makes it difficult, if not impossible, to control bleeding with replacement factor VIII therapy.

About hemophilia B

Hemophilia B is a genetic disorder caused by missing or defective factor IX, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a change in a gene.2
 
Current standard of care for patients with hemophilia B involves chronic replacement of FIX protein through intravenous infusion. In 2013, the World Federation of Hemophilia estimated there were more than 28,000 hemophilia B patients worldwide, including 4,000 patients in the United States.
 
 


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