Pharming announces US FDA approval of Joenja® (leniolisib) as the first and only treatment indicated for APDS
LEIDEN, The Netherlands, March 24, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) 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.
- 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 approval of a treatment option for one of the more than 450 primary immunodeficiencies is also a key moment for the broader primary immunodeficiency community.
- Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS.
- With the approval of Joenja®, as a treatment for a rare pediatric disease, the FDA granted Pharming a priority review voucher ("PRV").