FDA Accepts NDA for Leniolisib to Treat Children with APDS

The U.S. Food and Drug Administration (FDA) has accepted Pharming Group’s supplemental new drug application (NDA) for leniolisib to treat activated phosphoinositide 3-kinase delta syndrome, or APDS, in children aged 4 to 11 years. There are no approved treatments in this age group for APDS, a rare primary immunodeficiency caused by variants in PIK3CD or PIK3R1, which is vital to immune cell development and function.

Leniolisib (Joenja) is an oral, selective phosphoinositide 3-kinase delta inhibitor that inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, an important cellular messenger that regulates cell proliferation and differentiation, cytokine production, cell survival, angiogenesis and metabolism.

The supplemental NDA is based on data from a multinational, single-arm Phase III trial in children aged 4 to 11 years who experienced measurable reductions in lymphadenopathy and a statistically significant increase in naïve B cells over 12 weeks, indicating the underlying immune defect was being corrected. Also, the application included safety data from eight months of treatment.

“The study demonstrated clinically meaningful results in both efficacy endpoints and was generally safe and well-tolerated by the 21 patients,” said Anurag Relan, MD, chief medical officer at Pharming Group.

Patients experienced these improvements across all four dose levels in the study, Dr. Relan noted, adding that these findings were consistent with benefits seen in adolescent and adult patients as well. FDA approved leniolisib for patients aged 12 years and older in March 2023.

A Prescription Drug User Fee Act date was set for Jan. 31, 2026.

References

Gawel, R. FDA Accepts Supplemental New Drug Application for Leniolisib for Children with APDS. Healio, Oct. 20, 2025. Accessed at www.healio.com/news/allergy-asthma/20251019/fda-accepts-supplemental-new-drug-application-for-leniolisib-for-children-with-apds.

BSTQ Staff
BioSupply Trends Quarterly [BSTQ] is the definitive source for industry trends, news and information for the biopharmaceuticals marketplace. With timely and critical information, each themed issue covers topics ranging from product breakthroughs, industry insights and innovations, up-to-the-minute news on the latest clinical trials, accessibility, and service and safety concerns.