Note: The following is edited from a press release from PRNewswire.com. Read the full press release in it鈥檚 entirety聽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.聽 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,聽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聽(PERSEPT 1).聽 The study focused on the potential treatment of episodic bleeding events in adult and adolescent patients. 聽Two initial dose regimens were tested in 468 bleeding events occurring in 27 inhibitor patients.聽 Both study arms met the primary endpoint of hemostatic success (evidence of cessation of bleeding). 聽The majority (85%) of the bleeding events treated with the initial 225聽碌g/kg dose required no further therapy.聽 The Pain VAS (Visual Analog Scale) showed significant improvement, consistent with the efficacy findings. 聽The results were presented as a late-breaking oral abstract by Dr.聽Michael Wang, UC Denver in July at the World Federation of Hemophilia 2016 Congress held in聽Orlando, FL.