Press Release: Rilzabrutinib granted orphan drug designation in the US for sickle cell disease

Core Viewpoint - The FDA has granted orphan drug designation to rilzabrutinib for sickle cell disease, highlighting its potential to address unmet medical needs in rare diseases [1][2]. 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 [7]. Product Information - Rilzabrutinib is a novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that aims to restore immune balance through multi-immune modulation [5]. - The drug has received multiple orphan drug designations, including for immune thrombocytopenia (ITP), warm autoimmune hemolytic anemia (wAIHA), and IgG4-related disease (IgG4-RD) [2][8]. Disease Context - Sickle cell disease affects over 100,000 people in the US, predominantly impacting African American and Hispanic populations, leading to severe pain and reduced life expectancy [6]. - The disease is characterized by misshapen red blood cells that block blood flow, causing various health complications [6]. Regulatory Status - Rilzabrutinib is currently under regulatory review in the US, EU, and China for its potential use in ITP, with a target action date for FDA decision set for August 29, 2025 [3][8].