Leniolisib - (pediactric)

June 4, 2026: Pharming Group (Euronext: PHARM; NASDAQ: PHAR) said the FDA has accepted its resubmitted supplemental NDA for Joenja (leniolisib), an oral selective PI3Kδ inhibitor, seeking U.S. approval to treat children aged 4–11 years with activated phosphoinositide 3-kinase delta syndrome (APDS). The resubmission follows a Complete Response Letter dated January 30, 2026 and a Type A FDA meeting on March 26, 2026, and requests approval of 40 mg and 50 mg twice-daily dosing for pediatric patients weighing ≥27 kg. Pharming said the resubmission includes additional data requested by FDA related to analytical methods for production batch testing. The FDA assigned a PDUFA target action date of October 24, 2026.

Pharming also plans a separate sNDA in summer 2026 for lower-dose regimens for children weighing <27 kg (and notes a separate filing for lower-weight pediatric patients planned for 2H 2026).

The filing is supported by an open-label, multinational, single-arm Phase III study in 4–11-year-olds showing 12-week improvements in lymphadenopathy and naïve B cells; adverse events were mild-to-moderate, with no drug-related serious AEs and all patients completing 12 weeks. Joenja is already FDA-approved for APDS in patients ≥12 years (March 2023).