FDA approves Pharming’s Joenja for children

The US Food and Drug Administration (FDA) has approved Dutch biopharma firm Pharming’s (Euronext: PHARM) supplemental New Drug Application (sNDA) for Joenja (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.

With this approval, Joenja becomes the first FDA-approved treatment for children aged 4 to 11 years with APDS in the USA. The newly approved Joenja doses are expected to be available to eligible pediatric patients in the USA in October through Pharming’s established specialty distribution network and patient-support infrastructure.