wAIHA | Rilzabrutinib Receives FDA Breakthrough Therapy Designation
The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to rilzabrutinib, an investigational oral Bruton’s tyrosine kinase (BTK) inhibitor developed by Sanofi, for the treatment of warm autoimmune haemolytic anaemia (wAIHA).
Warm autoimmune haemolytic anaemia is a rare autoimmune disorder characterized by the premature destruction of red blood cells, leading to anemia and other serious complications, such as significant fatigue, dizziness, palpitations, and shortness of breath, and even potentially life-threatening complications such as thromboembolism.
The designation is supported by data from the Phase 2b LUMINA-2 study, which is evaluating the safety and efficacy of rilzabrutinib in patients with wAIHA, while a Phase 3 trial (LUMINA-3) is currently underway comparing the therapy with placebo.
Trial results demonstrated clinically meaningful improvements in haemoglobin levels and reductions in markers of haemolysis, with many patients achieving durable responses. The therapy also showed a manageable safety profile, highlighting its potential as a promising new treatment option for individuals living with wAIHA, a condition for which effective therapies remain limited.
Breakthrough Therapy designation is intended to accelerate the development and regulatory review of medicines for serious conditions when early clinical evidence indicates the potential for substantial improvement over existing therapies.
In parallel, Japan’s Ministry of Health, Labour and Welfare has granted Orphan Drug designation to rilzabrutinib for the same indication, highlighting the significant unmet medical need in wAIHA.
Rilzabrutinib is currently approved under the brand name Wayrilz™ for the treatment of adults with persistent or chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.
Source: Sanofi Press Release
