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Japan approves Joenja for treating APDS in patients aged 4 and older, first global approval for young children.

Market News
24 Mar 2026
Pharming Group N.V.
View Source
Bullish
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Japan's Ministry of Health has approved Joenja (leniolisib), an oral PI3K delta inhibitor, for treating activated PI3K delta syndrome (APDS) in patients aged 4 years and older. This marks the first treatment approved in Japan specifically for APDS and the first global approval for children aged 4 to 11 with the disease. Approval was based on positive Phase III clinical trial data showing significant improvements in immune function and disease symptoms. The drug is expected to launch in Japan after pricing agreement with the National Health Insurance system, offering a targeted treatment option for this rare immunodeficiency disorder.

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