CHMP backs Joenja for APDS, putting EU approval in sight

Pharming said the European Medicines Agency’s Committee for Medicinal Products for Human Use, or CHMP, has issued 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, Joenja would become the first approved treatment for APDS in the EU. The oral PI3Kδ inhibitor is already approved in the U.S. for patients 12 and older, in the U.K. for the same age group, and in Japan for adults and children ages 4 and older. Pharming said the CHMP opinion was supported by a randomized phase 3 trial in 31 patients and longer-term extension data in 37 patients with a median exposure of about three years. (stocktitan.net)

Why it matters: For veterinary professionals, this is another reminder that precision therapies for ultra-rare immune disorders are moving through global regulators even when patient populations are very small. APDS is a human disease, but the regulatory path is notable: Pharming has been building a worldwide footprint for leniolisib while also dealing with uneven label expansion, including a February 1, 2026, FDA Complete Response Letter for a U.S. supplemental filing in children ages 4 to 11. That split outcome underscores how manufacturing, age-range data, and regional review standards can shape access as much as clinical efficacy does. (pharming.com)

What to watch: The next step is a European Commission decision, which typically follows a positive CHMP opinion within about two months. (health.ec.europa.eu)