Sanofi has received regulatory approval in the US market for Wayrilz (rilzabrutinib) as the first BTK inhibitor for chronic immune thrombocytopaenia, demonstrating 23% durable platelet response versus 0% for placebo in the Phase III LUNA 3 trial. The reversible covalent inhibitor achieved platelet counts ≥50,000/μL in 64% of patients within 12 weeks, with median time to first response of 15 days. Sanofi acquired rilzabrutinib through its USD 3.7 billion purchase of Principia Biopharma in 2020.

The approval addresses critical unmet needs in this autoimmune bleeding disorder where platelet destruction and impaired production lead to counts below 100,000/μL. Rilzabrutinib's dual mechanism reduces pathogenic antibody production and macrophage-mediated platelet clearance. Sanofi has submitted a New Drug Application (NDA) in China for the same indication, potentially expanding access to this novel therapy for patients failing conventional treatments like corticosteroids and thrombopoietin receptor agonists.

According to PharmCube's NextBiopharm® database, 13 companies are developing a total of six BTK inhibitor programs in immune thrombocytopaenia. Click here to request a free trial for NextBiopharm®.