FDA approves leniolisib (Joenja) as the first treatment for APDS: A breaththrough in the field of immunology

Abstract

Activated PI3K delta Syndrome (APDS) is a rare genetic heterogeneous disorder initially identified in 2013. The primary cause of APDS is an immune imbalance leading to immune deficiency and dysregulation. Symptoms of APDS are diverse primarily including recurrent respiratory infections and increased predisposition to lymphoma. The disease is challenging to diagnose as patients are often misdiagnosed. Genetic testing can be used to make a confirmatory diagnosis. There were no FDA-approved treatments specifically targeting the root cause of APDS, PI3Kδ until the recent promising outcomes seen in the clinical trials of leniolisib. However, long term effects of the drug remain a concern as further research is required.