French healthcare company Sanofi S.A. (Euronext Paris:SAN) (Nasdaq:SNY) announced on Monday that the US Food and Drug Administration (FDA) has granted breakthrough therapy designation to Wayrilz (rilzabrutinib) for the treatment of warm autoimmune haemolytic anaemia (wAIHA), a rare autoimmune disorder causing red blood cell destruction.

The Japanese Ministry of Health, Labour and Welfare has also granted rilzabrutinib orphan drug status for the same condition.

Both designations are based on data from the ongoing LUMINA 2 phase 2b study, with LUMINA 3 phase 3 currently assessing rilzabrutinib against placebo. Rilzabrutinib is the first investigational BTK inhibitor for wAIHA and targets complex immune-system dysregulation through multi-immune modulation.

The therapy is approved in the United States, European Union, and United Arab Emirates for immune thrombocytopenia (ITP) under the brand name Wayrilz and is under review in Japan for ITP. Rilzabrutinib also holds multiple global regulatory designations for rare diseases, including orphan drug status in the US for autoimmune haemolytic anaemia, IgG4-related disease, and sickle cell disease, as well as FDA fast track and EU orphan designations. These designations underscore its broad therapeutic potential for serious and life-threatening conditions.