Pharming: European Green Light for Joenja, Launch in Germany in Q3 2026
The European Commission granted marketing authorization on Friday for Joenja (leniolisib), the first and only approved treatment in the European Union for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. The first European launch is expected in Germany in the third quarter of 2026.
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
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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.
SectorPharmacie et biotechnologies›Biotechnologies
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Context
Period
Period: T3 2026
Key reported figures
Revenue: 117,6 millions de dollars
Quarterly revenue: 117,6 millions de dollars
Revenue growth: 21 %
Guidance from the release
Tightened 2026 total revenue guidance to between US$380 million and US$390 million from prior US$375 million to US$395 million
Risks mentioned
Joenja revenue growth was partly driven by temporary benefit from ordering patterns in other international markets
RUCONEST third quarter revenue includes an early seasonal increase in channel inventory at a single U.S. specialty pharmacy customer, following significant drawdowns earlier in the year
Napazimone (KL1333): enrollment in the pivotal FALCON trial now expected to complete in the first half of 2027 (about 145 of 180 subjects by end-2026), with topline results approximately one year after completion of enrollment
Opportunities identified
RUCONEST stabilization and continued recovery of demand in the US (third quarter revenue of about US$93.8 million, +14% year-on-year and +30% versus the second quarter of 2026)
Joenja strong growth driven by launches in Germany, Japan and continued penetration in the U.K.
Substantial growth potential for Joenja with estimated CVID with immune dysregulation prevalence of 39 patients per million vs 1.5 for APDS
Outlook / guidance
Expected revenue: 385 millions de dollars
Management commentary: Tightened 2026 total revenue guidance to between US$380 million and US$390 million (1% to 4% growth) from prior US$375 million to US$395 million (0% to 5% growth)
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