Joenja (leniolisib) (children aged 4 years and older)

September 25, 2026: Pharming (NASDAQ: PHAR) said the FDA accepted and granted Priority Review to an sNDA for lower doses of Joenja (leniolisib) for activated phosphoinositide 3-kinase delta syndrome (APDS) in children aged 4 years and older weighing 13 kg to 27 kg. The PDUFA target action date is January 30, 2027.

The filing is supported by positive data from an open-label, multinational, single-arm Phase III study in children aged 4 to 11 years. Over 12 weeks, the study showed improvements in two clinically relevant hallmarks of APDS: reduced lymphadenopathy and increased naive B cells, which the company said indicate correction of the underlying immune defect. The submission also includes scientific and clinical pharmacology assessments supporting dosing in lower-weight pediatric patients.

The update follows the recent September 2026 U.S. approval expansion for Joenja in children aged 4 to 11 years weighing at least 27 kg. The press release also notes Joenja was first approved in March 2023 for adults and pediatric patients aged 12 years and older with APDS.