The US Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib for sickle cell disease (SCD).  Rilzabrutinib is an oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that works via multi-immune modulation, to target a reduction in vaso-occlusive crises, which may occur via inflammation.  BTK, expressed in B cells, macrophages, and other innate […]