Pharming announces US FDA approval of Joenja® (leniolisib) as the first and only treatment indicated for APDS
Pharming Group N.V. announces that the US Food and Drug Administration (FDA) has approved Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency.
The FDA evaluated the Joenja® application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Joenja® is expected to launch in the US in early April and will be available for shipment in mid-April.
Pharming held a conference call for investors and analysts on March 27, 2023 at 14:00 CEST/08:00 EST. Download the presentation slides and transcript below and rewatch the webcast.