Pharming Group N.V. announced that Japan’s Ministry of Health, Labour and Welfare approved Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in patients aged 4 years and older. This is the first approved treatment for APDS in Japan and the first global approval for children aged 4 to 11, with OrphanPacific, Inc. as the Marketing Authorization Holder.
Key takeaways
Japan’s MHLW approved Joenja® (leniolisib) on March 24, 2026, for APDS in patients aged 4 years and older.
First approved treatment in Japan specifically for APDS.
First global approval of Joenja for children aged 4 to 11 with APDS.
OrphanPacific, Inc. serves as Marketing Authorization Holder for Joenja in Japan and handles supply and distribution.
Approval based on Phase III data from global randomized study in patients 12+, Japanese study in patients 12+, and multinational pediatric study in children 4-11.
Launch expected following agreement with MHLW on National Health Insurance drug price.