Sanofi's Rilzabrutinib Receives Breakthrough Therapy Designation in US for Warm Autoimmune Hemolytic Anemia
The U.S. Food and Drug Administration (FDA) has granted Sanofi's rilzabrutinib (Wayrilz) breakthrough therapy designation for the treatment of warm autoimmune hemolytic anemia (wAIHA).
French pharmaceutical company Sanofi has received significant regulatory acknowledgments for its drug rilzabrutinib.
The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to rilzabrutinib, marketed as Wayrilz, in February 2026 for patients with warm autoimmune hemolytic anemia (wAIHA). Concurrently, Japan's Ministry of Health, Labour and Welfare designated rilzabrutinib as an orphan drug for the same condition.
These designations are supported by clinical data from the ongoing LUMINA 2 study. wAIHA is a rare autoimmune disorder characterized by the destruction of red blood cells, potentially leading to anemia, fatigue, and serious organ damage. Currently, there are no approved treatments that specifically target the underlying cause of this rare condition.
The FDA's breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions where preliminary evidence indicates the therapy may offer substantial improvement over available treatments. The orphan drug status in Japan is granted to medicines intended to address rare diseases with high unmet medical needs.
Rilzabrutinib is already approved in the U.S., EU, and UAE under the brand name Wayrilz for the treatment of adult immune thrombocytopenia (ITP). Its use for wAIHA is currently investigational.