FDA accepts the submitted Biologic License Application for Coagulation Factor VIIa (Recombinant), activated eptacog beta – Jan. 9, 2017
LOUISVILLE, Ky., Jan. 9, 2017 /PRNewswire/ — HEMA Biologics, LLC announced that the FDA has accepted the BLA for their first potential product for commercialization in the United States. The acceptance of the BLA for Coagulation Factor VIIa, Recombinant (eptacog beta activated), submitted by LFB, SA., represents the potential for a new treatment for Hemophilia A and B patients suffering with inhibitors.
“At HEMA Biologics, a company dedicated to bringing meaningful products and services to patients who suffer from rare bleeding disorders, we are very pleased that the FDA has accepted the submission for our lead product that, if approved, will provide a new treatment option for Hemophilia A and B patients with inhibitors,” commented P. Breckinridge Jones, CEO of HEMA Biologics.
The pivotal phase 3 trial supporting the license application is officially titled, Phase III 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 (PERSEPT 1). The study focused on the potential treatment of episodic bleeding events in adult and adolescent patients. Two initial dose regimens were tested in 468 bleeding events occurring in 27 inhibitor patients. Both study arms met the primary endpoint of hemostatic success (evidence of cessation of bleeding). The majority (85%) of the bleeding events treated with the initial 225 µg/kg dose required no further therapy. The Pain VAS (Visual Analog Scale) showed significant improvement, consistent with the efficacy findings. The results were presented as a late-breaking oral abstract by Dr. Michael Wang, UC Denver in July at the World Federation of Hemophilia 2016 Congress held in Orlando, FL.
All doses of eptacog beta were well-tolerated, with no detectable immunological or thrombotic responses to eptacog beta or host proteins. Two patients experienced treatment-emergent adverse events that were considered drug-related: peripheral venous hematoma and low-grade fever. One patient experienced a serious intracranial hemorrhage following an episode of tonsillitis after his treatment participation had ended.
About Eptacog Beta, Activated Eptacog Beta is an innovative recombinant form of human Factor VIIa. This new chemical entity was developed using LFB SA’s proprietary rPRO™ technology. Eptacog beta is currently under clinical development, and has not received commercial approval from any regulatory authority. If approved by the FDA, HEMA Biologics, LLC will have full commercialization rights for North America.
About HEMA Biologics, LLC HEMA Biologics is a privately-held biopharmaceutical company, located in Louisville, KY that is exclusively focused on meeting the needs of patients living with rare bleeding disorders, supporting the community that cares for them, and bringing meaningful products and services to the marketplace to help improve their daily lives. HEMA Biologics takes pride in the heritage of LFB, the manufacturer of our products.
About LFB, SA LFB Group (www.lfb.fr) is a multinational biopharmaceutical company that develops, manufactures, and markets medicinal products for the treatment of serious and often rare diseases in several major therapeutic fields, including Hemostasis, Immunology and Intensive Care. LFB is the leading manufacturer of plasma-derived medicinal products in France and 6th worldwide, and is also among the leading European companies for the development of new-generation medicinal products or treatments based on biotechnologies. LFB is pursuing a growth strategy that seeks to extend its international activities and develop innovative therapies. Today, LFB currently markets its products in more than 40 countries around the world with a global turnover of €502.4 million in 2015.