Pharming’s Joenja sNDA resubmission accepted by FDA for pediatric APDS expansion

Pharming Group (NASDAQ:PHAR) announced today that the U.S. Food and Drug Administration (FDA) has accepted its resubmitted supplemental New Drug Application (sNDA) for Joenja (leniolisib).

The application seeks to expand the indication for Joenja to include the treatment of children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.

The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of October 24, 2026.

This resubmission addresses concerns raised in a Complete Response Letter (CRL) received on January 30, 2026, which primarily requested additional analytical data regarding production batch testing and clarification on dosing for specific patient weight groups.

The current sNDA resubmission specifically seeks approval for 40 mg and 50 mg twice-daily dosing for pediatric patients weighing 27 kg or more.

Pharming has indicated that this dose range covers a meaningful proportion of the identified pediatric APDS population.

The company also noted plans to file a separate sNDA later this summer to address the lower-dose requirements for children weighing less than 27 kg.

The filing is supported by data from an open-label, multinational, single-arm Phase III clinical study.

The trial demonstrated that pediatric patients treated with Joenja experienced improvements in key hallmarks of APDS, including reduced lymphadenopathy and increased naïve B cell counts over a 12-week period.

Safety data from the study indicated that treatment-emergent adverse events were mild to moderate in nature, with no drug-related serious adverse events reported.

Joenja, a selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, is already approved in the U.S. for the treatment of APDS in adult and pediatric patients aged 12 years and older.

If the current application is successful, Joenja would become the first FDA-approved targeted therapy for the treatment of children under 12 living with this condition.