Joenja cleared for pediatric APDS in US

What this means

Activated PI3K delta syndrome (APDS) is a rare inherited immune disorder in which the immune system's B and T cells behave abnormally, leaving patients with repeated infections, swollen lymph nodes, and other complications from early childhood. Joenja (leniolisib) is Pharming's oral pill that specifically blocks the overactive enzyme (PI3Kδ) driving the disease. FDA first approved Joenja in March 2023 for adults and pediatric patients 12 and older. On September 11, 2026 the FDA approved a supplemental application that extends the label to children ages 4-11 who weigh at least 27 kg, along with pediatric-appropriate 40 mg and 50 mg twice-daily doses. Joenja becomes the first FDA-approved treatment for children ages 4-11 with APDS in the US. The expansion is supported by a multinational open-label single-arm Phase 3 study in the 4-11 population, which showed reduced lymph node enlargement and increased naïve B cells over 12 weeks - both markers of the underlying immune defect improving. Pharming expects the new pediatric doses to be available in the US in October 2026.