If approved, Joenja will be the first approved treatment in the U.S. for children with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency disorder.
The resubmission of the supplemental New Drug Application (sNDA) seeks approval for dosages of 40 mg and 50 mg twice daily.
A separate sNDA application for approval of lower dosages for pediatric patients is planned for the second half of 2026.
Leiden, 4 June 2026: Pharming Group (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM/Nasdaq: PHAR) announces that the U.S. Food and Drug Administration (FDA) has accepted the resubmitted supplemental New Drug Application (sNDA), seeking approval for Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor for the treatment of children aged 4 through 11 years with activated phosphoinositide 3-kinase δ syndrome (APDS), a rare primary immunodeficiency disorder.
Following the Complete Response Letter (CRL) received on 30 January 2026, and a subsequent Type A meeting with the FDA on 26 March 2026, the resubmission seeks approval for dosages of 40 mg and 50 mg twice daily for pediatric patients weighing 27 kg or more, who represent a meaningful proportion of the identified pediatric patient population. The resubmission includes additional data requested by the FDA regarding the analytical methods used for testing manufacturing batches. A decision is expected by 24 October 2026 under the Prescription Drug User Fee Act (PDUFA).
A separate sNDA application for approval of lower dosages for pediatric patients weighing less than 27 kg is planned for this summer.
Chief Executive Officer Fabrice Chouraqui commented:
“We are encouraged that the FDA has accepted our resubmission, bringing us one step closer to potentially making Joenja available to younger patients with APDS. These young patients and their families continue to face the significant burden of this rare disease, and there remains a clear need for an approved targeted treatment. We look forward to continuing to work with the FDA to make Joenja available to the broader pediatric APDS population as quickly as possible.”
The resubmission is supported by positive data from an open-label, multinational, single-arm Phase III study in children aged 4 through 11 years, in which improvements were demonstrated over 12 weeks in two clinically relevant features of APDS: reduced lymphadenopathy and an increase in naïve B cells, together indicating correction of the underlying immune defect. Improvements in lymphoproliferation and correction of the immunophenotype were observed across all dose levels studied and were consistent with the improvements previously reported in adolescent and adult patients. All treatment-emergent adverse events were reported as mild to moderate in nature. No drug-related serious adverse events were observed, and all patients completed the 12-week treatment period.
Joenja received FDA approval in March 2023 for the treatment of APDS in adult and pediatric patients aged 12 years and older.
