Pharming receives FDA approval for Joenja in children with APDS

The U.S. Food and Drug Administration (FDA) has approved Joenja (leniolisib) for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), provided they weigh at least 27 kg. This decision makes Joenja the first FDA-approved treatment for this specific pediatric age group.

The approval follows a multinational Phase 3 study which showed improvements in clinically relevant measures, including reduced lymphadenopathy and increased naïve B cells. The safety profile was reported to be consistent with previous Joenja experience, with treatment-emergent adverse events being mild to moderate.

Pharming expects the newly approved doses to be available in the U.S. through its distribution network in October. The company has also submitted a separate application for lower dosages intended for children in the same age group who weigh between 13 kg and 27 kg.