FDA Approves Emory-developed Hemophilia Drug
Reprinted with permission from Emory's Health Science Center Update, November 2014
Last month the Food and Drug Administration approved Obizur, a treatment for acquired hemophilia A that was originally developed by a research team led by Emory hematologist Pete Lollar, Hemophilia of Georgia Professor of Pediatrics. The Obizur technology was licensed by Emory in 1998 to startup company Octagen and was eventually brought to commercial availability by the pharmaceutical firm Baxter International. Hemophilia A is caused by a deficiency of clotting factor VIII and can be either inherited or acquired.
In acquired hemophilia A, the immune system is somehow provoked into making antibodies against factor VIII that inactivate it. Acquired hemophilia is a challenge for doctors to deal with because patients frequently present with severe, life threatening bleeding and also because it’s a surprise: patients do not have a previous personal or family history of bleeding episodes. Antibodies to factor VIII also can be a problem for approximately 30% of patients with inherited hemophilia.
Lollar's team developed a modified form of factor VIII, derived from the protein sequence of pigs, which is less of a red flag to the immune system.
The safety and efficacy of Obizur was evaluated in a clinical trial of 29 adults with acquired hemophilia A who received Obizur to treat a serious bleeding episode. Obizur received orphan drug designation by the FDA because the drug is intended for use in treatment of a rare disease or condition.
After licensing the technology from Emory, Octagen and a French partner, Ipsen Biopharm, pursued preclinical and clinical studies and completed a phase 2 clinical trial in 2006. Ipsen purchased the rights to the modified factor VIII in 2008 and developed a partnership with Inspiration Biopharmaceuticals in 2010. The pivotal phase 3 clinical trial took place between 2010 and 2013, and the rights to what became Obizur were bought by Baxter in 2013.—Quinn Eastman, from Lab Land