Pharming: FDA Accepts New Joenja Submission for Ages 4-11, PDUFA Set for October 24, 2026
Pharming Group announced on Thursday that the FDA has accepted its supplemental New Drug Application (sNDA) for Joenja (leniolisib) to treat children aged 4 to 11 with APDS, a rare primary immunodeficiency. This resubmission follows a negative response in January and subsequent dialogue with the US authority.
The application seeks approval for dosages of 40 mg and 50 mg twice daily for children weighing 27 kg or more, representing a significant portion of the target pediatric population. The FDA initially issued objections (Complete Response Letter) on January 30, 2026, before Pharming engaged in a Type A meeting with the agency on March 26. The resubmission incorporates additional data requested by the FDA regarding the analytical methods used for production batch testing.
Efficacy and Tolerance Demonstrated in Children
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The resubmission is based on the results of an open-label, multinational, single-arm phase III study conducted in children aged 4 to 11. This study demonstrated improvements after 12 weeks in two clinically relevant features of APDS: reduction in lymphadenopathy and increase in naive B lymphocytes, indicative of a correction of the underlying immune defect. All reported adverse events were classified as mild to moderate, with no serious drug-related adverse events reported. All patients completed the 12-week treatment period.
A Second Application Planned for Lighter Weight Children
Pharming is preparing a second sNDA, scheduled for the summer of 2026, to seek approval for lower doses intended for patients weighing less than 27 kg. The FDA has assigned a PDUFA (Prescription Drug User Fee Act) action deadline of October 24, 2026, for the current resubmission. Joenja received US approval in March 2023 for the treatment of APDS in adult and pediatric patients aged 12 and older.
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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