Biopharmaceutical company Pharming Group N.V. (Euronext Amsterdam:PHARM) (NASDAQ:PHAR) announced on Tuesday that Japan's Ministry of Health, Labour and Welfare has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adult and paediatric patients aged four years and older.

This decision marks the first approved therapy for the condition in Japan and the first globally to include children aged 4 to 11.

The approval is supported by Phase III clinical data, including multinational and Japanese studies demonstrating significant reductions in lymphadenopathy and improvements in immune function markers. The therapy was generally well tolerated, with commonly reported adverse reactions including headache, sinusitis, and atopic dermatitis.

Under a partnership agreement, OrphanPacific will serve as the marketing authorisation holder in Japan and oversee supply and distribution, with commercial launch expected following national health insurance pricing approval. The application was reviewed under a priority pathway after receiving orphan drug designation in 2023.

Joenja is already approved in the United States and the United Kingdom for patients aged 12 and older, while regulatory reviews are ongoing in the European Economic Area, Canada, and other markets.