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The European Medicines Agency (EMA) has granted orphan designation to rilzabrutinib, an investigational oral Bruton’s tyrosine kinase (BTK) inhibitor from Sanofi (1). The designation applies to the treatment of IgG4-related disease (IgG4-RD). Orphan designation is granted to therapies targeting rare, serious, or debilitating conditions affecting no more than five in 10,000 people in the European Union, underscoring the unmet medical need in this patient population (1).
Clinical data support potential in rare immune-mediated disorders
EMA’s decision is based on results from a Phase II clinical trial (NCT04520451) in which patients received…