By Fineline Cube 
France-based Sanofi (NASDAQ: SNY) announced that it has received an additional Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its oral Bruton’s tyrosine kinase (BTK) inhibitor rilzabrutinib. This designation is for the treatment of sickle cell disease and follows previous ODDs for immune thrombocytopenia (ITP), warm autoimmune hemolytic anemia (wAIHA), and IgG4-related disease (IgG4-RD).
Rilzabrutinib’s Mechanism and Potential
Rilzabrutinib is designed to reduce vascular occlusion crises in sickle cell disease patients by addressing inflammation and other underlying causes through immune regulation. Preclinical data demonstrated that rilzabrutinib reduced vaso-occlusion and inflammation in transgenic mice models of sickle cell disease.
Regulatory Review
The drug is currently under regulatory review in the US, EU, and China for its potential use in immune thrombocytopenia (ITP), highlighting its broad therapeutic potential.-Fineline Info & Tech