Sanofi has secured FDA Breakthrough Therapy Designation for its oral Bruton’s tyrosine kinase inhibitor, rilzabrutinib (Wayrilz), in the treatment of warm autoimmune hemolytic anemia (wAIHA). The designation, announced on February 9 2026, is intended to accelerate the drug’s development and review for this rare, life‑threatening condition.
Rilzabrutinib is a reversible BTK inhibitor that is already approved for immune thrombocytopenia (ITP). The FDA designation was based on data from the LUMINA 2 phase 2b study, which demonstrated clinically meaningful improvements in hemolysis markers and transfusion independence. A phase 3 LUMINA 3 trial is currently underway to further evaluate efficacy and safety.
wAIHA is a rare disorder with no approved targeted therapy; the breakthrough designation signals that rilzabrutinib may offer a substantial improvement over existing corticosteroid‑based treatments. The designation also opens the possibility of accelerated approval, potentially bringing a first‑in‑class oral therapy to patients sooner.
The designation strengthens Sanofi’s rare‑disease portfolio, which now includes 86 projects across four therapeutic areas. It positions rilzabrutinib as a key asset in the company’s strategy to expand treatment options for rare autoimmune disorders.
Sanofi’s recent financial performance underscores the strategic importance of this milestone. Full‑year 2024 sales reached EUR 41,081 million with a net income of EUR 5,560 million, while full‑year 2025 sales climbed to EUR 43,626 million and net income rose to EUR 7,813 million, reflecting robust growth that supports continued investment in rare‑disease development.
The Japanese Ministry of Health, Labour and Welfare has also granted orphan drug status to rilzabrutinib for wAIHA, further supporting its development in a high‑need market and reinforcing Sanofi’s commitment to rare diseases.
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