CHMP backs Pharming’s Joenja for APDS in the EU

Pharming said the European Medicines Agency’s Committee for Medicinal Products for Human Use, or CHMP, has adopted a positive opinion recommending approval of Joenja (leniolisib) for activated phosphoinositide 3-kinase delta syndrome, or APDS, in adults and children ages 12 and older. If the European Commission follows that recommendation, as it often does, Joenja would become the first approved treatment for APDS in the European Union, with a final decision expected in the second quarter of 2026. The recommendation is based on Phase II/III data in 31 patients and longer-term extension data in 37 patients, according to Pharming. (live.euronext.com)

Why it matters: APDS is an ultra-rare primary immunodeficiency driven by overactive PI3Kδ signaling, and EU clinicians have had no authorized disease-specific therapy to use, relying instead on supportive care such as immunoglobulin replacement and infection management. For specialists following rare immune disorders, a centralized EU authorization would widen access across all 27 member states plus Norway, Iceland, and Liechtenstein, and could further raise awareness of a condition that is often misdiagnosed and associated with long diagnostic delays. (ema.europa.eu)

What to watch: The next step is the European Commission’s final decision, expected within about two months of the March 27, 2026 CHMP opinion. (live.euronext.com)