Biopharmaceutical company Pharming Group N.V. (Euronext Amsterdam:PHARM) (NASDAQ:PHAR) announced on Wednesday that the US Food and Drug Administration (FDA) has accepted its supplemental New Drug Application for leniolisib in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).

The application has been granted Priority Review with a PDUFA target action date of 31 January 2026.

Pharming's submission is based on positive results from a multinational Phase III study showing improvements in lymphadenopathy and naïve B cell counts over 12 weeks, indicating correction of the underlying immune defect. Safety data from eight months of treatment were also included.

If approved, leniolisib would become the first and only treatment indicated for children under 12 with APDS globally. The therapy is marketed in the United States as Joenja and received FDA approval in March 2023 for patients aged 12 years and older.