LEIDEN, Netherlands — September 25, 2026

Pharming Group N.V. announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for Priority Review seeking approval of lower doses of Joenja® (leniolisib) for children aged 4 years and older weighing 13 kg to 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS). The FDA has assigned a PDUFA target action date of January 30, 2027. If approved, the supplemental application would expand access to Joenja among smaller pediatric patients who are currently outside the U.S. product’s approved weight range.

Lower-Dose Application Supported by Pediatric Phase 3 Data

The sNDA is supported by data from an open-label, multinational, single-arm Phase 3 study in children aged 4 to 11 years, which evaluated clinically relevant manifestations of APDS over 12 weeks. The study reported improvements in lymphadenopathy and naive B-cell levels, two measures reflecting immune dysregulation and immune-cell development in APDS. Pharming also submitted additional scientific and clinical pharmacology assessments supporting the proposed lower dosing regimen for pediatric patients weighing between 13 kg and 27 kg. The submitted evidence is intended to establish an appropriate dose for smaller children while maintaining the therapeutic exposure associated with leniolisib treatment.

Joenja Targets PI3Kδ Pathway in APDS

Joenja is an oral, selective PI3Kδ inhibitor designed to inhibit the PI3Kδ signaling pathway, which is dysregulated in APDS due to pathogenic variants in PIK3CD or PIK3R1. APDS is a rare primary immunodeficiency characterized by immune dysfunction and dysregulation that can include recurrent respiratory infections, lymphoproliferation, autoimmunity and enteropathy. Joenja was first approved by the FDA in March 2023 for adults and pediatric patients aged 12 years and older with APDS. In September 2026, the FDA expanded the U.S. approval to children aged 4 to 11 years weighing at least 27 kg, while the current sNDA seeks to address lower-weight pediatric patients beginning at 13 kg.

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Pharming Seeks Broader Pediatric Access to Joenja

The Priority Review provides a regulatory pathway for Pharming to potentially extend Joenja treatment to a broader pediatric APDS population in the United States. FDA Priority Review is intended for applications for medicines that, if approved, may provide significant improvements in the treatment, prevention or diagnosis of serious conditions. Leniolisib is also approved for APDS in several other markets and remains under regulatory review in additional countries. Pharming is evaluating leniolisib in two Phase 2 studies involving primary immunodeficiencies with immune dysregulation, although its safety and efficacy in these conditions outside APDS have not been established. The FDA’s January 30, 2027 PDUFA date will determine whether the proposed lower-dose regimen can be added to the U.S. Joenja label for eligible children weighing 13 kg to 27 kg.

Source :Pharming press release