Sanofi(US:SNY) Globenewswire·2026-02-09 06:00Core Viewpoint - Sanofi's rilzabrutinib has received breakthrough therapy designation from the FDA and orphan drug designation in Japan for treating warm autoimmune hemolytic anemia (wAIHA), highlighting the urgent need for effective treatments for this rare autoimmune disorder [1][3][4]. Group 1: Designations and Studies - The FDA's breakthrough therapy designation aims to expedite the development of medicines for serious conditions, indicating rilzabrutinib may show substantial improvement over existing treatments [3]. - Rilzabrutinib is currently being evaluated in the LUMINA 2 phase 2b study and the new LUMINA 3 phase 3 study, which compares rilzabrutinib with placebo for wAIHA patients [2]. - There are no approved treatments specifically targeting the underlying causes of wAIHA, which can lead to severe health complications [2]. Group 2: Rilzabrutinib Overview - Rilzabrutinib is a novel oral, reversible Bruton's tyrosine kinase (BTK) inhibitor, already approved for immune thrombocytopenia (ITP) in the US, EU, and UAE, and under review in Japan [4][8]. - The drug addresses immune system dysregulation through multi-immune modulation, making it a potential treatment for various rare diseases [7][8]. - Rilzabrutinib has received multiple regulatory designations, including orphan drug status for autoimmune hemolytic anemia and other rare diseases [5][9]. Group 3: About wAIHA - Warm autoimmune hemolytic anemia is a rare and potentially life-threatening condition, affecting 4 to 24 individuals per 100,000 in the US and EU, and 3 to 10 per million in Japan [6]. - Symptoms include fatigue, dizziness, and serious complications like thromboembolism, emphasizing the need for effective treatments [6]. Group 4: Company Overview - Sanofi is an R&D-driven biopharma company focused on improving lives through innovative medicines and vaccines, with a commitment to addressing urgent healthcare challenges [10].