Pharming Group has announced that the US Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for leniolisib in paediatric patients.
Leniolisib is an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, proposed for the treatment of children aged four to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS). The therapy, marketed under the brand name Joenja, received FDA approval in 2023 for patients aged 12 years and older.
The FDA has granted the sNDA Priority Review and set a Prescription Drug User Fee Act (PDUFA) target action date of 31 January 2026. Pharming noted that Priority Review status reflects the lack of approved treatment options for children under 12 with APDS, a condition that typically manifests in early childhood. APDS is characterised by immune dysregulation and recurrent infections, and may lead to permanent lung damage and lymphoma.
The application is supported by data from a multinational, single-arm phase 3 study in patients aged four to 11 years. Results showed clinical improvements, including reduced lymphadenopathy and increased naïve B cells, which indicated the underlying immune effect of the condition being corrected
“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 US living with APDS,” said Fabrice Chouraqui, CEO of Pharming.