Leniolisib was approved by the FDA as the first treatment of activated phosphoinositide 3kinase Delta syndrome in both adults and children 12 years old and older.
A press release by the FDA stated that leniolisib (Joenja Pharming Group), was evaluated for efficacy in a 12-week, blinded, randomized and placebo-controlled study in which 31 patients aged 12 and older were tested. The trial included 31 adults and children with confirmed activated phosphoinositide 3kinase Delta syndrome (APDS)-associated genetic PI3Kd (with a documented variant in either PIK3CD (or PIK3R1). Researchers looked at improvement in
