Pharming Group: Joenja® Treatment Approved in Japan, a Historic First
Pharming Group announces the approval by the Japanese Ministry of Health, Labour and Welfare of Joenja® (leniolisib), a selective inhibitor of phosphoinositide 3-kinase delta, for the treatment of APDS in patients aged 4 years and older. This approval marks a first in Japan and is the first specific targeted treatment for this rare primary immunodeficiency approved in the country.
Joenja becomes the first approved treatment in Japan specifically for APDS and the first globally approved treatment for children aged 4 to 11 years with this condition. The approval encompasses both adult and pediatric patients aged 4 years and older. The product is already approved and marketed in the United States and the United Kingdom for patients aged 12 years and older. The Japanese Pharmaceutical Agency (PMDA) reviewed the application through a priority assessment procedure following the orphan drug designation granted in May 2023.
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OrphanPacific, Inc. serves as the marketing authorization holder for Joenja in Japan and collaborates with Pharming for the supply and distribution of the product. The launch of Joenja is anticipated following an agreement with the MHLW on the pricing of the drug under the national health insurance. The approval is based on data from Pharming's Phase III clinical trial program, including a randomized placebo-controlled study in patients aged 12 years and older, an open Japanese study, as well as positive data from a multinational open Phase III study in children aged 4 to 11 years.
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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