Rare Blood Disorders

Some of the most serious unmet patient needs today are in the field of hematology, and we are committed to significantly improving the health and lives of people with rare blood disorders around the world through groundbreaking science.

With our acquisitions of Bioverativ and Ablynx in 2018, and our own in-house programs, we have the opportunity to apply our legacy of a patient-centered approach with our commitment to innovation and rigorous clinical research to make a meaningful difference for people affected by rare blood disorders around the world.

Our Rare Blood Disorders franchise is positioned for growth with an unmatched hemophilia portfolio, the first approved treatment for acquired thrombotic thrombocytopenic purpura (aTTP), and the potential to launch several more therapies over the next five years, providing the opportunity to bring new therapies to patients who currently have few options.

Inspired by Patients


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.

16,500
people helped since 2015
117,000
acute bleeds treated
1,800
surgeries made possible through the World Federation of Hemophilia Humanitarian Aid Program


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.

Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Beginning in 2018, we launched the first approved treatment for acquired thrombotic thrombocytopenic purpura (aTTP), a rare, life-threatening, autoimmune-based, blood disorder characterized by extensive clot formation in small blood vessels throughout the body, leading to a very low platelet count, a loss of red blood cells and restricted blood supply to parts of the body.

Poised for Growth

In addition to our current portfolio, we have a strong pipeline focusing on areas of unmet need in the rare blood disorder community, including hemophilia, cold agglutinin disease, sickle cell disease, and beta thalassemia.

  • Cold Agglutinin Disease
    We are currently conducting late-stage clinical trials of a breakthrough investigational therapy for the treatment of cold agglutinin disease (CAD), a chronic and severe rare blood disorder with no approved treatments.
  • Sickle Cell Disease and Beta Thalassemia
    In partnership with Sangamo, we are developing gene-edited cell therapies with the aim of providing a one-time, lasting treatment for people living with sickle cell disease or beta thalassemia. These lifelong rare blood disorders are triggered by genetic mutations. Currently, patients have few treatment options.

Learn about treatment options:

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