Sanofi Wins FDA Breakthrough Tag for Wayrilz in wAIHA

Sanofi has secured an FDA breakthrough therapy designation for Wayrilz (rilzabrutinib) to treat adults with warm autoimmune hemolytic anemia (wAIHA), a rare autoimmune disease that destroys red blood cells.

The relocation has been a positive development to the patients who have not yet been placed under the approved treatments that help in directly overcoming the cause of the condition. Rilzabrutinib has also received orphan designation by the Ministry of Health, Labour and Welfare in Japan in regard to wAIHA.

This choice can be justified by the results of the current LUMINA 2 phase 2b trial (NCT05002777), which is examining the drug safety and its effectiveness in patients with wAIHA. A confirmatory phase 3 (NCT07086976) LUMINA 3 randomized trial comparing rilzabrutinib and placebo is currently in progress.

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An FDA breakthrough therapy designation assists to accelerate the designing and regulatory approval of medicines in a serious or life-threatening illness in situations where preliminary clinical results show a meaningful improvement compared to current care. The orphan designation of Japan is a type that is applied to treatments that target high unmet need and rare diseases.

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

Wayrilz is licensed in the US, the EU and UAE in immune thrombocytopenia (ITP) and under regulatory process in Japan in ITP. Its application in wAIHA and other rare disorders is still experimental.