PARIS (dpa-AFX) - French drug major Sanofi S.A. (SNYNF,SNY) announced Tuesday that the US Food and Drug Administration has granted orphan drug designation to rilzabrutinib for sickle cell disease. This is the fourth orphan drug designation for rilzabrutinib in rare diseases.
Sickle cell disease is a group of rare, genetic blood disorders in which red blood cells are misshapen, typically in a sickle or crescent shape, causing them to get stuck in small blood vessels, blocking blood flow. The affected people will have episodes of severe pain as well as other health complications including infections, stroke, lung, eye, and kidney disease. In the U.S., sickle cell disease affects more than 100,000 people, approximately 90% of whom are African American and 3-9% of whom are Hispanic or Latino.
Rilzabrutinib is a novel, advanced, oral, reversible Brutons tyrosine kinase or BTK inhibitor that has the potential to be an effective new medicine for several rare immune-mediated or inflammatory diseases by working to restore immune balance via multi-immune modulation.
Rilzabrutinib works through multi-immune modulation to target a reduction in vaso-occlusive crises, which may occur through inflammation, in sickle cell disease.
The company had presented preclinical data on sickle cell disease at ASH 2024 showing that rilzabrutinib helped reduce vaso-occlusion blockage of blood vessels and inflammation in transgenic mice with sickle cell disease.
The FDA grants orphan drug designation to investigational therapies addressing rare medical diseases or conditions that affect less than 200,000 people in the US.
In addition to sickle cell disease, rilzabrutinib has received orphan drug designation for immune thrombocytopenia (ITP) in the US, the EU, and Japan, for warm autoimmune hemolytic anemia (wAIHA) in the US and the EU, and for IgG4-related disease (IgG4-RD) in the US.
Karin Knobe, Global Head of Development, Rare Diseases, said, 'Receiving our fourth orphan drug designation for rilzabrutinib reinforces our continued dedication to developing medicines to address the unmet medical needs of people living with rare diseases. People with sickle cell disease often live with severe episodes of pain from vaso-occlusive crises and other complications that can significantly impact both quality of life and life expectancy. There remains a need for novel treatment approaches to address these experiences by modulating the immune system responses that can contribute to sickle cell disease pathogenesis.'
Sanofi added that the safety and efficacy of rilzabrutinib have not been determined by any regulatory authority, while it is currently under regulatory review in the US, the EU, and in China for its potential use in ITP.
The target action date for the FDA regulatory decision for ITP, which was granted fast track designation, is August 29, 2025.
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