Pharming Group Obtains US FDA Nod for Rare Primary Immunodeficiency Drug Joenja

11:37 PM EDT, 09/13/2026 (MT Newswires) -- Pharming Group (PHARM.AS) secured US Food and Drug Administration approval for Joenja, or leniolisib, as a treatment for the rare primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome, or APDS, among children aged four to 11 years, particularly those weighing at least 27 kilograms. The biotechnology company said Sept. 11 that it supported the supplemental new drug application with positive data from a multinational, open-label, and single-arm phase 3 trial for the approved indication. Pharming is also seeking FDA approval for Joenja as a treatment for APDS among pediatric patients in the same age group but weighing between 13 kilograms and less than 27 kilograms. Previously, the regulator approved the drug for patients aged at least 12 years.