Leniolisib: First Approval.

Leniolisib (JOENJA) is an oral selective phosphoinositide 3-kinase-delta (PI3Kδ) inhibitor being developed by Pharming Group NV in-licensed from Novartis for the treatment of immunodeficiency disorders. In March 2023, leniolisib received its first approval for the treatment of activated PI3Kδ syndrome (APDS) in adult and paediatric patients 12 years of age and older. Leniolisib is also under regulatory review in European Union for the treatment of APDS. Development of leniolisib for the treatment of Sjögren's syndrome has been discontinued. This article summarizes the milestones in the development of leniolisib leading to this first approval for APDS.