Pharming wins FDA Priority Review for Joenja in lighter children

Pharming's sNDA for lower leniolisib doses in children weighing 13-27 kg with APDS has a PDUFA date of 30 January 2027.

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Pharming has received FDA acceptance and Priority Review for a supplemental New Drug Application covering lower doses of Joenja (leniolisib) in children aged four and older who weigh between 13 kg and 27 kg and have activated phosphoinositide 3-kinase delta syndrome (APDS). The agency has set a PDUFA target action date of 30 January 2027.

The filing extends Joenja's approved paediatric reach. The FDA first approved leniolisib for adults and patients aged 12 and older in March 2023, then in September 2026 expanded the label to children aged 4 to 11 weighing at least 27 kg. This sNDA, if approved, would bring in the smallest patients currently excluded from treatment: children who fall below that weight threshold.

Clinical basis

The supplemental application is supported by data from an open-label, multinational, single-arm Phase 3 study in children aged 4 to 11. Over 12 weeks, the study recorded reductions in lymphadenopathy and increases in naive B cells, two biomarkers considered indicative of correction of the underlying immune defect in APDS. Pharming also included additional clinical pharmacology assessments to justify the proposed dosing in lower-weight patients.

Anurag Relan, Chief Medical Officer at Pharming, said the acceptance "brings us closer to the possibility of reaching smaller children who currently are ineligible for treatment with Joenja," and said the company would work with the FDA to make the medicine available "as efficiently as possible."

Disease and competitive context

APDS is caused by gain-of-function variants in PIK3CD or PIK3R1, genes critical to PI3K-delta pathway regulation, leading to immune cell maturation failure, recurrent sinopulmonary infections, lymphoproliferation, and autoimmunity. The condition affects approximately one to two people per million worldwide and carries a reported median diagnostic delay of seven years, by which point progressive organ damage, including permanent lung injury and increased lymphoma risk, may already have occurred.

Leniolisib is positioned by Pharming as the first and only approved targeted therapy for APDS, giving it a degree of regulatory exclusivity in an ultra-rare indication where no direct small-molecule competitor has yet received approval. The broader PI3K inhibitor class has faced a difficult period in oncology, with several approved agents withdrawn or restricted due to safety concerns, but the APDS setting involves a genetically defined patient population with a clear mechanistic rationale for PI3K-delta inhibition, which has so far supported a cleaner tolerability profile.

Priority Review designation, granted by the FDA to applications that could offer significant improvements for serious conditions, reduces the standard review clock from twelve months to six. That designation, combined with the January 2027 PDUFA date, suggests a potential decision in the first quarter of next year. Pharming has indicated leniolisib is also under review in several additional countries and is being evaluated in Phase 2 trials for other primary immunodeficiencies with immune dysregulation, though the company has not yet established safety or efficacy beyond APDS in those studies.

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