Pharming Group announces U.S. FDA acceptance and Priority Review of supplemental New Drug Application forleniolisib in children with APDS aged 4 to 11 years
October 1, 2025 – Biotechnology, Drug Discovery, Other, Pharmaceutical – APDS, FDA, Pharming Group, new drug application, priority review
- If approved, leniolisib will be first and only treatment indicated for children with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency
- Decision based on positive data from multinational Phase III study in children
aged 4 to 11 years with APDS - PDUFA target action date of January 31, 2026.
1 October 2025 — Leiden, the Netherlands — Pharming Group N.V. today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) seeking approval for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 31, 2026.
The sNDA submitted to the FDA is based on positive data from the multinational, single-arm Phase
III study in children aged 4 to 11 years, which showed improvements over 12 weeks in two clinically
relevant hallmarks of the condition, reduced lymphadenopathy and increased naïve B cells,
together indicating a correction of the underlying immune defect. The submission also included
safety data from 8 months of treatment.
The FDA grants Priority Review to applications for medicines that, if approved, would offer
significant improvements in effectiveness or safety of the treatment, prevention, or diagnosis of
serious conditions.1 There are no approved treatments for children with APDS under the age of 12
years globally.
Fabrice Chouraqui, Chief Executive Officer of Pharming, commented: “APDS is a rare, complex, and progressive primary immunodeficiency. Typically, it begins in early childhood causing immune dysregulation, recurrent infections and potentially permanent lung damage and lymphoma. Early access to targeted therapies has the potential to change the trajectory of the disease for young patients. Today’s Priority Review designation marks a significant step for children aged 4-11 in the U.S. living with APDS.”
Leniolisib, marketed under the brand name Joenja® in the U.S., received approval from the U.S. FDA
for the treatment of APDS in adult and pediatric patients 12 years of age and older in March 2023.
About Pharming Group N.V.
Pharming Group N.V. is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. We are developing and commercializing a portfolio of innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, with a significant proportion of its employees based in the U.S. For more information, visit www.pharming.com.
