Operational updates
Pharming Group receives positive CHMP opinion for Joenja® (leniolisib) for the treatment of APDS in adult and pediatric patients 12 years and older
Pharming Group N.V. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adult and pediatric patients 12 years of age and older.
- If approved, Joenja® (leniolisib) would become the first approved treatment in the European Union for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency
- Decision based on Phase II/III clinical data demonstrating statistically significant impact on measures of immune dysregulation and immunodeficiency
- Final European Commission decision expected in Q2 2026