Rare Blood Disorders



Inspired by Patients

Some of our patients’ most serious 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.

Our Focus

Our Rare Blood Disorders franchise is positioned for growth with a hemophilia portfolio, an approved treatment in Europe 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.

Hemophilia

Hemophilia is a rare genetic disorder in which a person’s blood does not clot normally because of insufficient levels of certain clotting factors.  People with hemophilia may bleed spontaneously or for a prolonged period of time after an injury due to deficient clotting (1).   While hemophilia is most often an inherited condition, almost a third of people diagnosed do not have a family history.  There are an estimated 400,000 people worldwide living with hemophilia, however, 75% do not have access to adequate treatment or care.  Hemophilia does not discriminate – all races and ethnic groups are affected (2).

Our first innovative medicines to treat hemophilia were launched in 2014.  Since then, we have developed a hemophilia portfolio with 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 for people living 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, an international rare diseases company, we have pledged the single largest donation of hemophilia factor therapy in history: up to one billion International Units (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.

Infographic on world federation of hemophilia humanitarian aid program

Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

aTTP is 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, loss of red blood cells and restricted blood supply to parts of the body.  It can result in widespread organ damage especially in the brain and heart.

In 2018, in Europe, we launched the first approved treatment for aTTP, bringing a new treatment option to patients who previously had none. In the United States, this treatment was approved by the FDA in February 2019. 

Cold Agglutinin Disease (CAD)

CAD is a severe, chronic, blood disorder that  results in the immune system mistakenly destroying red blood cells. People with CAD suffer from chronic and severe anemia due to the high rate of red blood cell destruction. In addition to anemia, people affected by CAD are more likely to experience stroke and heart attack as a result of blood clots in arteries, and deep vein thrombosis and pulmonary embolism as a result of blood clots in the veins.

We are currently conducting late-stage clinical trials of an investigational therapy for the treatment of cold agglutinin disease (CAD), which has no approved treatments today.

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. 

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