Pharming Group Secures U.S. FDA Priority Review For sNDA of Leniolisib In Children Aged 4–11 With APDS

Pharming Group N.V. today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor. The application seeks approval for the use of leniolisib in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. The FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 31, 2026.

The submission is supported by results from a multinational, single-arm Phase III clinical trial conducted in children within this age group. Over a 12-week period, the study demonstrated clinically meaningful improvements in two key features of APDS: reduced lymphadenopathy and increased naïve B cells. Together, these findings indicate a correction of the underlying immune defect. Additionally, the application included safety data from eight months of treatment in pediatric patients.

Fabrice Chouraqui, Chief Executive Officer of Pharming, stated, “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.”

The FDA grants Priority Review to applications for medicines that, if approved, could provide significant improvements in the treatment, prevention, or diagnosis of serious conditions. At present, there are no approved therapies available globally for children under 12 years of age living with APDS. Leniolisib is already marketed in the United States under the brand name Joenja®. In March 2023, the FDA approved Joenja® for the treatment of APDS in adult and pediatric patients aged 12 years and older. This latest application aims to expand the use of leniolisib to younger children who currently have no approved treatment options.