Pharming: European Green Light for Joenja, Launch in Germany in Q3 2026

Approval Based on Randomized Clinical Trials

The European Commission's approval is based on the results of a randomized, triple-blind, placebo-controlled phase II/III clinical trial involving 31 patients with APDS aged 12 and older. The study demonstrated statistically significant improvements in markers of dysregulation and immunodeficiency. The dossier also included long-term open-label extension data from 37 patients who had been receiving leniolisib for a median duration of three years. Joenja is an oral selective inhibitor of phosphoinositide 3-kinase delta that directly targets the underlying pathophysiology of the disease by inhibiting the production of phosphatidylinositol-3-4-5-trisphosphate, a cellular messenger that regulates proliferation, differentiation, and cytokine production.

Expanding Access Across Europe Following Previous Authorizations

The approval is valid in the 27 member states of the European Union, as well as in Norway, Iceland, and Liechtenstein. Joenja has received authorizations in the United States, the United Kingdom, Japan, Australia, and Israel; it is currently marketed in the United States and the United Kingdom for patients aged 12 and older (in Japan, the authorization covers patients from 4 years old). The launch in Germany in the third quarter of 2026 will be followed by other European launches pending the completion of national reimbursement negotiations. Leverne Marsh, Chief Commercial Officer at Pharming, stated that APDS is a progressive disease that has a profound and lasting impact on the quality of life of patients and their families, highlighting the urgent need for targeted treatment options.