Posted by Michael Wonder on 09 Feb 2026
Sanofi’s rilzabrutinib designated breakthrough therapy in the US and orphan drug in Japan for the treatment of warm autoimmune haemolytic anaemia
9 February 2026 - The US FDA has granted a designation as breakthrough therapy to Wayrilz (rilzabrutinib), a novel oral, reversible Bruton’s tyrosine kinase inhibitor, for the treatment of patients with warm auto-immune haemolytic anaemia, a rare autoimmune disorder marked by the destruction of red blood cells.
The Japanese Ministry of Health, Labour and Welfare has also provided rilzabrutinib an orphan designation for the same condition.
Both designations are based on clinical data from the ongoing LUMINA 2 pPhase 2b study assessing the efficacy and safety of rilzabrutinib for patients with warm autoimmune haemolytic anaemia.
