The FDA approved Joenja, also known as leniolisib, for treating activated phosphoinositide 3-kinase delta syndrome in children aged 4 to 11 years weighing at least 27 kg, according to an agency notice.
The notice highlighted that this is the first treatment approved for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency caused by variants in PIK3CD or PIK3R1, in this age group.
For patients with APDS aged 12 years and older, 70 mg Joenja (Pharming) oral tablets are taken twice daily per the FDA’s approval in March 2023. Joenja oral tablets are also taken