Posted by Michael Wonder on 25 Mar 2023
FDA approves first treatment for activated phosphoinositide 3-kinase delta syndrome
24 March 2023 - The FDA has approved Joenja (leniolisib) tablets as the first treatment for activated phosphoinositide 3-kinase delta syndrome in adult and paediatric patients 12 years of age and older.
The efficacy of Joenja was evaluated in the placebo-controlled portion of Study 2201, a 12‑week blinded, randomised, placebo-controlled study of 31 adult and paediatric patients 12 years of age and older with confirmed APDS associated genetic PI3Kδ mutation, with a documented variant in either PIK3CD or PIK3R1.
