Press Release: Sanofi's rilzabrutinib earns orphan designation in the EU for IgG4-related disease

Core Viewpoint - Sanofi's rilzabrutinib has received orphan designation from the European Medicines Agency for the treatment of IgG4-related disease, highlighting its potential in addressing rare and debilitating conditions [1][3]. Group 1: Rilzabrutinib's Designation and Studies - Rilzabrutinib has been granted orphan designation for IgG4-related disease (IgG4-RD) in the EU, which is a rare condition affecting no more than 5 in 10,000 persons [1] - A phase 2 study (NCT04520451) demonstrated that rilzabrutinib treatment for 52 weeks resulted in reduced disease flare and other disease markers, while also allowing for glucocorticoid sparing [2] - The safety profile of rilzabrutinib in the study was consistent with previous studies, with no new safety signals observed [2] Group 2: Regulatory Status and Designations - Rilzabrutinib has received orphan designations for immune thrombocytopenia (ITP) in the US, EU, and Japan, as well as for warm autoimmune hemolytic anemia, IgG4-RD, and sickle cell disease in the US [3] - The drug is currently under regulatory review in the US, EU, and China for its potential use in ITP, with a target action date for the US FDA decision set for August 29, 2025 [4] Group 3: Mechanism and Disease Overview - Rilzabrutinib is a novel oral, reversible covalent BTK inhibitor that aims to restore immune balance through multi-immune modulation, targeting BTK expressed in various immune cells [5] - IgG4-RD is a chronic immune-mediated rare disease that can lead to organ damage and affects approximately eight out of 100,000 adult patients in the US each year [6] Group 4: 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]