FDA Grants Breakthrough Designation for Wayrilz to Treat Autoimmune Hemolytic Anemia

The U.S. Food and Drug Administration (FDA) has granted a designation as breakthrough therapy to Wayrilz (rilzabrutinib), a novel oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor, for the treatment of patients with warm autoimmune hemolytic anemia (wAIHA), a rare autoimmune disorder marked by the destruction of red blood cells. 

The designation is based on clinical data from the ongoing LUMINA 2 Phase IIb study (NCT05002777) assessing the efficacy and safety of rilzabrutinib for patients with wAIHA. In addition, the new LUMINA 3 Phase III study (NCT07086976) is assessing rilzabrutinib compared with placebo in patients with wAIHA. There is currently no approved treatment that specifically targets the underlying cause of this rare autoimmune condition, which can lead to anemia, fatigue and serious organ damage.

“These recognitions highlight the critical unmet need that persists for people living with wAIHA,” said Karin Knobe, global head of development, rare diseases, at Sanfoi. “Furthermore, receiving such designations reinforces our commitment to advancing innovative medicines for rare diseases that currently have limited or no approved treatment options.”