Sanofi has announced that rilzabrutinib (Wayrilz) has received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) and Orphan Drug designation from Japan’s Ministry of Health, Labour and Welfare for the treatment of warm autoimmune hemolytic anemia (wAIHA), a rare and potentially life-threatening autoimmune blood disorder.
Rilzabrutinib is the first and only investigational Bruton’s tyrosine kinase inhibitor (BTKi) to receive FDA Breakthrough Therapy status for wAIHA. Both regulatory decisions are supported by clinical data from the ongoing LUMINA-2 Phase 2b study, which is evaluating the drug’s efficacy and safety in patients with wAIHA. A Phase 3 trial (LUMINA-3) is also underway, comparing rilzabrutinib with placebo in this patient population.
wAIHA is caused by immune-mediated destruction of red blood cells, leading to anemia, fatigue, and potentially serious complications, including organ damage and thromboembolism. There are currently no approved therapies that directly target the underlying disease mechanism.
FDA Breakthrough Therapy designation is intended to accelerate the development and review of treatments for serious conditions where early clinical evidence suggests substantial improvement over existing options. Japan’s Orphan Drug designation supports the development of therapies for rare diseases with high unmet medical need.
Rilzabrutinib is a novel oral, reversible covalent BTK inhibitor designed to restore immune balance through multi-immune modulation. BTK plays a central role in immune signaling pathways involved in several autoimmune and inflammatory diseases.
The drug is already approved in the US, European Union, and the UAE for the treatment of immune thrombocytopenia (ITP) and is currently under regulatory review for ITP in Japan.
Beyond wAIHA, rilzabrutinib holds multiple global regulatory designations across rare diseases, including IgG4-related disease and sickle cell disease, underscoring its broader therapeutic potential.
