Sanofi’s treatment granted orphan designation for rare chronic inflammatory condition
Ella Day | August 14, 2025 | News story | Medical Communications, Research and Development | European Alliance of Associations for Rheumatology, European Medicines Agency, IgG4-related disease, Rheumatology, Sanofi
The European Medicines Agency has granted orphan designation to Sanofi’s investigational Bruton’s tyrosine kinase (BTK) inhibitor, rilzabrutinib, for the treatment of IgG4-related disease (IgG4-RD), a chronic inflammatory condition. The designation is given to therapies targeting rare, serious or debilitating conditions affecting no more than five in 10,000 people in the EU.
The decision follows positive results from Sanofi’s phase 2 study, presented at the 2025 European Alliance of Associations for Rheumatology Congress in Barcelona, Spain in June. Over 52 weeks, rilzabrutinib reduced disease flares, improved disease markers and allowed for reduced glucocorticoid use in patients with IgG4-RD. The treatment’s safety profile was consistent with earlier trials, with no new safety concerns identified.
Rilzabrutinib has also received orphan designations for immune thrombocytopenia (ITP) in the US, EU and Japan, and for warm autoimmune haemolytic anaemia, IgG4-RD and sickle cell disease in the US. In addition, it has US fast track status for both ITP and IgG4-RD. Regulatory reviews for ITP are currently ongoing in the US, EU and China, with a US FDA decision expected on 29 August 2025.
IgG4-RD is a rare, chronic immune-mediated condition that can cause widespread organ damage. It affects an estimated eight in 100,000 adults in the US annually.
Ella Day
14/8/25
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