Note: The following is edited from a press release from PRNewswire.com. Read the full press release in it’s entirety here.
HEMA Biologics, LLC announced that the FDA has accepted the Biologics License Application(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.
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.