Rare Blood Disorders

Tucker, Hemophilia B, US

Some of the most serious unmet patient needs today are in the field of hematology, and Sanofi Genzyme is committed to breaking barriers through groundbreaking science to significantly improve the health and lives of people with rare blood disorders around the world.

In 2018, the Rare Blood Disorders franchise was created with two strategic acquisitions [Bioverativ and Ablynx] and in-house programs with the goal of fundamentally redefining the management of these rare and often chronic diseases. In 2020, Sanofi Genzyme expanded its pipeline with the acquisition of Principia, broadening its work in immune-mediated blood disorders. 

Hemophilia, a rare genetic blood disorder that impairs the ability of blood to clot, is the cornerstone of the Rare Blood Disorders franchise. Sanofi Genzyme’s extended half-life factor replacement therapies for people with hemophilia A and B were launched in 2014, becoming the first innovations in hemophilia management in 20 years. Since then, they have been used to treat over 3,500 people with hemophilia worldwide. Sanofi Genzyme has a steadfast commitment to the hemophilia community and is continuing to explore potential new treatment approaches which could allow patients to live beyond the limitations of their condition.

Building on its legacy of innovation for patients with limited treatment options, Sanofi Genzyme launched the first approved treatment for acquired thrombotic thrombocytopenic purpura (aTTP), a rare, life-threatening, autoimmune-based blood disorder. aTTP is characterized by extensive clot formation in small blood vessels throughout the body, leading to a low platelet count, a loss of red blood cells, and restricted blood supply to parts of the body.

Inspired by patients and driven by science, we are focused on helping to make a transformative difference in the standard of care for people affected by rare blood disorders around the world.

Inspired by patients worldwide
Stephan, hemophilia, Kenya
Jenny, acquired thrombotic thrombocytopenic purpura, US
Brad, cold agglutinin disease, Canada


Above from left to right: Stephan, hemophilia, Kenya (photo courtesy of the World Federation of Hemophilia); Jenny, acquired thrombotic thrombocytopenic purpura, US; Brad, cold agglutinin disease, Canada.

Research & Development

Sanofi continues to innovate with the goal of making a meaningful impact in the lives of people with rare blood disorders.

Humanitarian Program

Together with Sobi, Sanofi Genzyme has committed the single largest donation of hemophilia factor therapy in history.

Pipeline

Sanofi has one of the most robust rare blood disorders pipelines in the industry.


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