Rare Blood Disorders

Some of the most serious unmet patient needs today are in the field of hematology, and we are 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, we created our Rare Blood Disorders franchise with two strategic acquisitions [Bioverativ and Ablynx] and in-house programs with a goal of addressing unmet needs and fundamentally redefining the management of these rare and often chronic diseases. 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. 

Hemophilia, a rare genetic blood disorder that impairs the ability of blood to clot, is the cornerstone of our Rare Blood Disorders franchise. Our extended half-life factor replacement therapies for people with hemophilia A and B were the first innovations in hemophilia management in 20 years when launched in 2014 and have since been used to treat over 3,500 people with hemophilia worldwide. We have a steadfast commitment to the hemophilia community and are continuing to explore potential new treatment approaches which could allow patients to live a more active life.

Building on our legacy of innovation for patients with limited treatment options, we launched the first approved treatment for 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, a loss of red blood cells and restricted blood supply to parts of the body. 

Inspired by Patients

Stephan, hemophilia, Kenya
Jenny, aTTP, United States
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, USA; Brad, cold agglutinin disease, Canada.

Breaking Barriers Through Groundbreaking Science

We continue to innovate with the goal of making a meaningful impact in the lives of people with rare blood disorders. By harnessing the power of science and pursuing rigorous clinical research, we are charting a bold new course in the treatment of hemophilia, immune thrombocytopenic purpura and sickle cell disease and beta thalassemia. 

Hemophilia 
We have an unmatched hemophilia pipeline of investigational therapies designed to break through existing treatment limitations and address serious unmet needs for patients. 
We are exploring two potentially transformative approaches to hemophilia management in late-stage clinical trials. We are investigating a potential treatment designed to provide monthly protection with a subcutaneous prophylactic dose for people with hemophilia A or B, with and without inhibitors. Also, in partnership with Sobi, we are advancing a potential therapy with the aim to provide high sustained factor activity in a once-weekly treatment for people with hemophilia A. 

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.

Immune Thrombocytopenic Purpura
Immune thrombocytopenic Purpura (ITP) is a rare autoimmune disease where the body destroys healthy platelets leading to excessive bruising and bleeding. Clinical programs evaluating two investigational therapies to treat this disease are underway.

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.

A Commitment to Patients Around the World

We are guided by a deep appreciation and understanding of what it means to live with a rare blood disorder, and we learn by listening to the community – patients, caregivers, physicians, and advocacy groups. We believe our responsibility goes beyond developing therapies and realize innovation is only valuable if it benefits the patients that need it most. Sanofi is committed to empowering patients and their families through ongoing education and support programs that help people obtain access to our treatments.

As part of our steadfast commitment to the hemophilia community, we are helping to transform hemophilia care in the developing world together with Sobi and the World Federation of Hemophilia (WFH).

In June 2020, together with Sobi, Sanofi extended its support of the WFH Humanitarian Aid Program with an additional donation of up 500 million IUs of clotting factor over up to five years, fulfilling the 2014 pledge to donate up to one billion IUs of clotting factor for humanitarian use.

This donation, the single largest of its kind, is an important first step toward providing a predictable and sustainable supply of therapy to those most in need. Since donations began in 2015, the impact has been far-reaching.

450m IUs 
of clotting factor have treated over 17,200 people in 42 countries
>160,000
acute bleeds treated
>2,300
surgeries have taken place, including those that were life- and limb-saving 

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