Press Release: Rilzabrutinib granted orphan drug designation in the US for two rare diseases with no approved medicines

Rilzabrutinib granted orphan drug designation in the US for two rare diseases with no approved medicines Designation granted for warm autoimmune hemolytic anemia and IgG4-related diseaseRilzabrutinib is currently under regulatory review in the US, EU and China for potential use in immune thrombocytopenia  Paris, April 3, 2025. The US Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib, an investigational, novel, advanced, oral, reversible Bruton’s tyrosine kinase (BTK) in ...