The FDA has granted breakthrough therapy designation to Sanofi’s Wayrilz (rilzabrutinib) for warm autoimmune hemolytic anemia, making it the first and only investigational BTK inhibitor for this indication to receive the designation. Simultaneously, Japan’s Ministry of Health, Labour and Welfare granted orphan drug designation for the same condition.
Rilzabrutinib is an oral, reversible Bruton’s tyrosine kinase inhibitor that addresses immune-system dysregulation through multi-immune modulation. It is already approved in the U.S., EU, and UAE for immune thrombocytopenia. Warm autoimmune hemolytic anemia is a rare, potentially life-threatening autoimmune disorder affecting approximately 4 to 24 per 100,000 people in the U.S. and EU.
Clinical trials currently evaluating the drug include the Phase 2b LUMINA 2 and Phase 3 LUMINA 3 studies. Rilzabrutinib has accumulated multiple regulatory designations globally, including FDA orphan designations for autoimmune hemolytic anemia, IgG4-related disease, and sickle cell disease, as well as EU orphan designations for several indications.