FDA approves first treatment for children aged 4-11 years with APDS, a rare genetic disorder of the immune system

FDA has approved Joenja (leniolisib) tablets as the first treatment for activated phosphoinositide 3-kinase delta syndrome in children 4-11 years who weigh at least 27 kg. Joenja was first approved in 2023 for the same indication in patients 12 years and older at a dose of 70 mg orally twice a day.