Key Takeaways Sanofi's rilzabrutinib receives FDA orphan drug designation for SCD, its fourth such indication. Preclinical data show rilzabrutinib reduced vaso-occlusion and inflammation in SCD mouse models. Rilzabrutinib is also under FDA review for ITP, with a decision expected by August 29, 2025.Sanofi (SNY) announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, for sickle cell disease (SCD). The candidate exerts its effects through multi-immune ...

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].

Rilzabrutinib granted orphan drug designation in the US for two rare diseases with no approved medicines Designation granted for warm autoimmune hemolytic anemia and IgG4-related diseaseRilzabrutinib is currently under regulatory review in the US, EU and China for potential use in immune thrombocytopenia  Paris, April 3, 2025. The US Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib, an investigational, novel, advanced, oral, reversible Bruton’s tyrosine kinase (BTK) in ...