FDA Approves Pharming's Joenja (Leniolisib) as First Treatment for Children Aged 4-11 With Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS); U.S. Launch October 2026
Pharming Group N.V. (NASDAQ: PHAR) announced Friday September 11, 2026 that the FDA approved the company's supplemental new drug application (sNDA) for Joenja (leniolisib, an oral selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor) at 40 mg and 50 mg twice-daily dosing for children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS). Joenja becomes the first FDA-approved treatment for children aged 4-11 with APDS, a rare primary immunodeficiency driven by gain-of-function mutations in the PIK3CD or PIK3R1 genes producing constitutive PI3Kδ pathway activation and recurrent sinopulmonary infections. Approval follows the February 2026 complete response letter, which required additional supportive data. The newly approved doses will be available through Pharming's specialty distribution network and patient-support infrastructure starting October 2026. Joenja was originally approved in March 2023 for APDS patients aged 12 and older; the pediatric label expansion extends the treated population substantially.