SNY's Rilzabrutinib Wins 4th Orphan Drug Tag for Sickle Cell Disease

Key Takeaways Sanofi's rilzabrutinib receives FDA orphan drug designation for SCD, its fourth such indication. Preclinical data show rilzabrutinib reduced vaso-occlusion and inflammation in SCD mouse models. Rilzabrutinib is also under FDA review for ITP, with a decision expected by August 29, 2025.Sanofi (SNY) announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, for sickle cell disease (SCD). The candidate exerts its effects through multi-immune ...