FDA Approves Pharming's Immune Disorder Drug, As First APDS Therapy

The FDA approved Pharming Group N.V.'s PHAR Joenja (leniolisib) for 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 U.S. for APDS, a rare and progressive primary immunodeficiency.
The FDA evaluated the Joenja application for APDS under Priority Review. Joenja is expected to launch in the US in early April and will be available for shipment in mid-April.
Related: Pharming Highlights Interim Data From Rare Immune Deficiency Study.
With the approval of Joenja as a treatment for a rare pediatric disease, the FDA granted Pharming a priority review voucher ("PRV"). Under Pharming's 2019 exclusive license agreement with Novartis AG NVS for leniolisib.
Novartis has the right to purchase the PRV from Pharming for a small minority share of the value of the PRV.
Under the agreement, Pharming will make milestone payments to Novartis and another party for the approval and first commercial sale for APDS totaling $10.5 million and agreed to make certain additional milestone payments to Novartis in an aggregate amount of up to $190 million upon the achievement of certain leniolisib sales milestones.
Price Action: PHAR shares are down 12.60% at $13.08 during the premarket session on the last check Monday.