Above from left to right: Hakob, hemophilia, Armenia (photo courtesy of the World Federation of Hemophilia); Rosie, cold agglutinin disease, Canada; Tucker, hemophilia B, USA
A Steadfast Commitment to the Hemophilia Community
We are focused on advancing science to address the serious unmet needs of people living with hemophilia. Launched in 2014 as the first innovations in hemophilia treatment in nearly two decades, our extended half-life therapies offer proven protection for people with hemophilia A and B, and already have been prescribed to more than 3,500 people in the United States alone.
Today, we continue to innovate with the goal of making a meaningful impact in the lives of people with rare blood disorders. We have an unmatched hemophilia portfolio that includes a pipeline of investigational therapies designed to address the serious unmet needs of people living with hemophilia. We are currently in a late-stage clinical trial exploring an investigational treatment designed to provide targeted protection with monthly subcutaneous dosing for people with hemophilia A or B, with and without inhibitors.
Committed to the discovery and development of new medicines, we also have a novel factor VIII therapy in early clinical development for people with hemophilia A.
A Commitment to Patients Around the World
We are guided by a deep appreciation and understanding of what it means to live with hemophilia, and we learn by listening to the community – patients, caregivers, physicians, and advocacy groups.
Globally, we are helping to lead the transformation of hemophilia treatment in the developing world, where people with severe hemophilia often don’t survive to adulthood. Together with Sobi, we have pledged the single largest donation of hemophilia factor therapy in history: up to one billion IUs of clotting factor over 10 years, beginning with up to 500 million IUs to the World Federation of Hemophilia Humanitarian Aid Program over a period up to five years. This is an important first step toward providing a predictable and sustainable supply of therapy to those most in need.