• FDA approval expands Joenja indication to include children with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) ages 4 to 11 who weigh at least 27kg
  • Newly approved Joenja doses are expected to be available in October

Leiden, the Netherlands, September 11, 2026: Pharming (NASDAQ:PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for 40 mg and 50 mg twice-daily dosing of Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. With this approval, Joenja becomes the first FDA-approved treatment for children aged 4 to 11 years with APDS in the U.S. The newly approved Joenja doses are expected to be available to eligible pediatric patients in the U.S. in October through Pharming’s established specialty distribution network and patient-support infrastructure.

On July 30, Pharming also submitted a separate sNDA seeking approval of lower Joenja doses for pediatric APDS patients aged 4 to11 years who weigh 13 kg to less than 27 kg.