Rare Blood Disorders

Jenny, living with acquired thrombotic thrombocytopenic purpura, U.S.

Sanofi significantly improves the health and lives of people with rare blood disorders around the world through groundbreaking science.

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

Sanofi’s Rare Blood Disorders franchise is positioned for growth with a great hemophilia portfolio, the first approved treatment for acquired thrombotic thrombocytopenic purpura (aTTP), and the potential to launch several prospective first-in-class therapies over the next five years, providing the opportunity to bring 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 dedication to the hemophilia community

Building on its existing hemophilia portfolio, Sanofi is focused on advancing science with the goal of making a meaningful impact in the lives of people with hemophilia. It is currently conducting a late-stage clinical trial exploring an investigational treatment designed to provide extended protection across all hemophilia indications.

Committed to the discovery and development of new medicines, Sanofi  also has a novel factor VIII therapy in early clinical development for people with hemophilia A that has the potential to provide high sustained factor activity levels with once weekly dosing.

A commitment to patients around the world

Sanofi is 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.

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


Globally, Sanofi is 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, Sanofi continues to deliver on the 2014 pledge to donate up to one billion international units (IUs) of clotting factor over ten years, beginning with up to 500 million IUs to the World Federation of Hemophilia Humanitarian Aid Program over a period of up to five years. 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.

aTTP

In 2018, Sanofi launched the first approved treatment for aTTP, a rare, life-threatening, autoimmune-mediated blood disorder characterized by clot formation in small blood vessels throughout the body, leading to a very low platelet count, restricted blood supply to parts of the body, and ultimately organ damage.

Poised for growth

In addition to our current portfolio, Sanofi has a strong pipeline focusing on disease areas where new treatment options are needed, including hemophilia, cold agglutinin disease (CAD), sickle cell disease, and beta thalassemia.

CAD
We are currently conducting late-stage clinical trials of a breakthrough investigational therapy for the treatment of CAD, a chronic and serious autoimmune hemolytic anemia with no approved therapy.

Sickle cell disease and beta thalassemia
In partnership with Sangamo, Sanofi is 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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