India, June 3 -- 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 ...