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FDA approves Joenja for children aged 4 to 11 with APDS, establishing first treatment for this pediatric population
PHARPharming Group N.V.
The U.S. FDA approved a supplemental New Drug Application for Joenja (leniolisib), expanding its indication to treat children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS). This approval establishes Joenja as the first FDA-approved treatment for this pediatric population. Newly approved doses are expected to be available in October through Pharming’s specialty distribution network. A separate submission for lower doses for patients weighing 13 kg to less than 27 kg was filed on July 30, 2026. The approval is based on Phase III study data demonstrating improvements in lymphadenopathy and naïve B cells. Pharming Group N.V. is a global biotechnology company that develops and commercializes innovative medicines for rare immune and genetic diseases.