CHMP backs Joenja for APDS, setting up EU decision
Pharming has moved a step closer to expanding Joenja into the European Union after CHMP adopted a positive opinion recommending marketing authorization for leniolisib in adults and pediatric patients ages 12 and older with activated phosphoinositide 3-kinase delta syndrome. The opinion marks an important turn in a review that had been stalled despite regulators already agreeing the drug’s clinical benefit and safety were favorable. Pharming said it expects a final European Commission decision in Q2 2026. (stocktitan.net)
The backstory matters here. In May 2024, Pharming disclosed that CHMP’s remaining concern was not about whether leniolisib worked, but about a chemistry, manufacturing, and controls requirement tied to starting materials in the manufacturing process. CHMP asked the company to complete that work before approval and granted an extension to January 2026. At the time, Pharming said the committee had affirmed the drug’s positive clinical benefit and safety, echoing the view of an ad hoc expert group. (pharming.com)
Joenja already has a regulatory foothold outside the EU. The FDA approved it in March 2023 as the first treatment for APDS in adults and children 12 and older. In England and Wales, NICE later recommended it for reimbursement and NHS use in the same age group. Pharming has also pointed to broader geographic expansion, including approval in Australia in March 2025 and in Japan in March 2026, according to company materials and recent company-linked coverage. (fda.gov)
The clinical package behind the filing is small, but that’s expected in an ultra-rare disease. FDA materials say efficacy was evaluated in a 12-week blinded, randomized, placebo-controlled study of 31 patients with confirmed APDS, with co-primary endpoints focused on lymphoproliferation and normalization of naïve B cells. Company-linked reporting on the CHMP opinion says the recommendation was also supported by long-term open-label extension data in 37 patients with a median of three years of exposure. APDS itself is estimated to affect around 1 to 2 people per million and is associated with recurrent sinopulmonary infections, immune dysregulation, enlarged lymphoid tissue, and elevated lymphoma risk. (fda.gov)
Independent expert reaction to this specific CHMP opinion was limited in public sources at the time of writing, but the broader clinical context helps explain why the decision is notable. APDS is often described as an inborn error of immunity with a long diagnostic odyssey, and recent literature has highlighted the burden of delayed diagnosis and multisystem disease. That means regulatory progress for a targeted therapy tends to matter beyond market expansion alone, especially in specialist immunology centers that are trying to move patients from supportive care toward pathway-directed treatment. (frontiersin.org)
Why it matters: For veterinary professionals tracking translational medicine, rare disease regulation, or comparative immunology, this is a reminder of how targeted therapies for genetically defined immune disorders are moving through global regulators even when datasets are necessarily small. The EU review also shows how nonclinical issues, especially manufacturing and CMC questions, can delay access long after efficacy and safety appear settled. In practical terms, a positive EC decision would give European clinicians another approved option for a disease that has historically relied on symptom management, immune support, and case-by-case escalation. (pharming.com)
There’s also a second regulatory thread to watch. While the EU filing concerns patients 12 and older, Pharming’s U.S. effort to expand Joenja into children ages 4 to 11 hit a setback in February 2026, when the FDA issued a complete response letter asking for more pediatric pharmacokinetic data and clarification around a manufacturing-related observation. The existing U.S. approval for patients 12 and older was unaffected. That split outcome underscores that even with momentum in one region, pediatric expansion can hinge on dosing and exposure questions. (pharming.com)
What to watch: The immediate next step is the European Commission decision expected in Q2 2026; after that, attention will likely shift to launch timing, country-level access and reimbursement, and whether Pharming can resolve the U.S. pediatric questions for children ages 4 to 11. (stocktitan.net)