CHMP backs Pharming’s Joenja for APDS in the EU

Pharming has moved a step closer to expanding Joenja into the European Union after the EMA’s CHMP issued a positive opinion for leniolisib in activated phosphoinositide 3-kinase delta syndrome, or APDS, for adults and pediatric patients ages 12 and older. The company said a European Commission decision is expected in Q2 2026, and approval would make Joenja the first authorized treatment for APDS in the EU. (live.euronext.com)

The opinion follows a longer and more complicated EU review than Pharming had originally hoped. In 2024, the company disclosed that CHMP had asked for additional manufacturing-related information during its review of the marketing authorization application, pushing the timeline into 2026. Pharming later said it expected a CHMP opinion in the first half of 2026, and that forecast has now materialized. (pharming.com)

Joenja is already on the market in several countries, which gives some context for the EU filing. The FDA approved the drug in March 2023 for APDS in patients 12 and older, the U.K. authorized it in September 2024, NICE recommended it for NHS use in England and Wales in April 2025, and Japan approved it on March 24, 2026, including a broader label covering patients ages 4 and older. (pharming.com)

The CHMP recommendation is based on a multinational, triple-blind, placebo-controlled, randomized Phase II/III trial in 31 patients ages 12 and older, plus long-term open-label extension data in 37 patients with a median of three years of treatment exposure, according to Pharming. A peer-reviewed subgroup analysis published in Clinical Immunology reported that leniolisib was well tolerated in a 12-week placebo-controlled Phase III study and met both co-primary endpoints, including reduction in lymph node burden and improvement in naïve B-cell percentages, measures tied to immune dysregulation and immunodeficiency in APDS. (live.euronext.com)

Company commentary has centered on unmet need. Pharming CEO Fabrice Chouraqui said the positive opinion reflects the strength of the clinical data and the need for a treatment that targets the underlying cause of disease. That framing is consistent with the broader regulatory history: the EMA’s orphan designation materials noted that no satisfactory treatments were authorized in the EU for APDS at the time of designation, with care focused mainly on controlling infections, including immunoglobulin replacement therapy. (live.euronext.com)

Why it matters: For clinicians, this is less about a routine label expansion and more about the possible arrival of the first disease-targeted option for a very small, frequently overlooked patient population. APDS was first characterized in 2013, can present with recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy, and is often missed for years before genetic testing confirms the diagnosis. Pharming has said patients can face a median seven-year diagnostic delay, and that the disease’s progression can lead to permanent lung damage and lymphoma. In practice, any EU approval could sharpen referral pathways, increase demand for genetic workups in suspected primary immunodeficiency, and create new conversations around earlier diagnosis and long-term monitoring. (pharming.com)

There’s also a broader commercial and development angle. While Europe may be nearing a decision for patients 12 and older, Pharming hit a setback in the U.S. pediatric expansion effort in February 2026, when the FDA issued a complete response letter for children ages 4 to 11, citing the need for additional pharmacokinetic data in lower-weight patients and more information on one analytical batch-testing method. Japan’s recent approval in patients 4 and older shows regulators are not moving in lockstep, and it suggests Pharming’s next challenge will be harmonizing evidence packages and manufacturing responses across markets. (pharming.com)

What to watch: The immediate milestone is the European Commission decision expected by Q2 2026; after that, attention will likely shift to launch timing in EU member states, reimbursement negotiations, and whether Pharming can resolve the FDA’s pediatric questions to broaden access in younger children. (live.euronext.com)