Rilzabrutinib granted orphan drug designation for sickle cell disease in the US

Recently the US FDA granted orphan drug designation to rilzabrutinib, a reversible Bruton’s tyrosine kinase inhibitor that works via multi-immune modulation, to target a reduction in vaso-occlusive crises, which may occur via inflammation, in sickle cell disease.

Karin Knobe, MD, PhD
Global Head of Development, Rare Diseases
“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.”

Nonetheless, rilzabrutinib has received orphan drug designation for immune thrombocytopenia in the US, the EU, and Japan, for warm autoimmune hemolytic anemia in the US and the EU, and for IgG4-related disease in the US.

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Related Topics and Keywords

rilzabrutinib, Sanofi, Sickle Cell Disease