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SNY's Rilzabrutinib Gets FDA's Orphan Drug Tag for Two Rare Diseases
SanofiSanofi(US:SNY) ZACKSยท2025-04-03 16:30

Core Viewpoint - Sanofi's investigational BTK inhibitor, rilzabrutinib, has received orphan drug designation from the FDA for treating warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD), addressing significant unmet medical needs in these rare diseases [1][2]. Company Developments - Rilzabrutinib is currently undergoing mid-stage studies for both wAIHA and IgG4-RD, with no approved therapies available for these conditions [1]. - Data from a phase IIb study for wAIHA indicated clinically meaningful outcomes in response rates and disease markers [5]. - A phase IIa study for IgG4-RD patients showed a reduction in disease flare and other disease markers after 52 weeks of treatment, with further details to be announced at an upcoming medical conference [6]. Regulatory Status - A regulatory application for rilzabrutinib to treat immune thrombocytopenia (ITP) is under review in the United States, with a decision expected on August 29, 2025. The drug has received both fast-track and orphan drug designations for ITP [8]. - Rilzabrutinib is also under review in the European Union and China for ITP treatment [9]. Market Performance - Year-to-date, Sanofi's shares have increased by 11.9%, outperforming the industry average rise of 0.8% [3].