LEIDEN, Netherlands — September 11, 2026

Pharming Group N.V. announced that the U.S. Food and Drug Administration (FDA) has approved a supplemental New Drug Application (sNDA) expanding Joenja® (leniolisib) to children aged 4 to 11 years who weigh at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS). The approval makes Joenja the first FDA-approved treatment for children aged 4 to 11 with APDS in the United States, expanding access to the company’s oral, selective PI3Kδ inhibitor. The newly approved 40 mg and 50 mg twice-daily doses are expected to become available to eligible U.S. pediatric patients in October 2026 through Pharming’s established specialty distribution and patient-support infrastructure. The company has also submitted a separate sNDA seeking approval of lower doses for children weighing 13 kg to less than 27 kg, extending its strategy to address a broader pediatric APDS population.

Phase 3 Data Support Pediatric Joenja Approval

The FDA decision is supported by data from a multinational, open-label, single-arm Phase 3 study in children aged 4 to 11 years with APDS. Over 12 weeks, treatment with Joenja demonstrated improvements in two clinically relevant characteristics of the disease: reduced lymphadenopathy and increased naïve B cells. Pharming said the findings together indicate improvement in the underlying immune defect associated with APDS, rather than addressing symptoms alone. The safety profile observed in the pediatric study was consistent with previous Joenja experience, with all treatment-emergent adverse events reported as mild to moderate and no drug-related serious adverse events observed. Joenja was previously approved by the FDA in March 2023 for adults and pediatric patients aged 12 years and older with APDS. The latest approval therefore represents a significant expansion of the product’s U.S. label and provides a regulatory foundation for earlier intervention in children with the rare immune disorder.

Joenja Targets the Underlying PI3Kδ Pathway

APDS is a rare primary immunodeficiency caused by variants in PIK3CD or PIK3R1 that result in excessive activity of the PI3Kδ pathway and impaired immune-cell development and function. The disease commonly emerges during childhood and can cause recurrent sinopulmonary infections, lymphoproliferation, autoimmunity and enteropathy, with progressive complications including permanent lung damage and lymphoma. Pharming estimates that APDS affects approximately 1 to 2 people per million worldwide, while diagnosis can be delayed because its symptoms overlap with other immune disorders. Joenja is an oral small-molecule PI3Kδ inhibitor designed to inhibit production of phosphatidylinositol-3-4-5-trisphosphate, an important cellular signaling messenger involved in immune-cell proliferation, differentiation, cytokine production and survival. By targeting the overactive pathway, leniolisib is intended to address the underlying immune dysregulation associated with APDS. The pediatric approval strengthens Pharming’s positioning in rare immune and genetic diseases while expanding the potential treatment window earlier in the disease course.

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Pharming Builds Global Pediatric Rare-Disease Access

The U.S. pediatric approval strengthens Pharming’s broader strategy to expand Joenja access across age groups and international markets. Joenja is already approved for APDS in the United States, United Kingdom, Australia, Israel and the European Union for patients aged 12 years and older, while Japan has approved the treatment for patients aged 4 years and older. Leniolisib is also under regulatory review in Canada and several other countries, and Pharming is evaluating the therapy in Phase 2 studies involving primary immunodeficiencies with immune dysregulation beyond APDS, although safety and efficacy have not been established for those conditions. The company’s immediate U.S. focus will be making the newly approved pediatric doses available through its specialty distribution network and patient-support infrastructure. By extending Joenja into younger children while pursuing additional weight-based dosing and international regulatory opportunities, Pharming is expanding the commercial and clinical reach of its lead rare-disease therapy and strengthening its position in pediatric immunology.

Source: Pharming press release