FDA Accepts Joenja sNDA for Lower-Weight Children With APDS and Grants Priority Review – Pharming
Pharming announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) seeking approval for lower doses of Joenja (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, for children aged 4 years and older who weigh between 13 kg and 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.
The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2027.
The sNDA is supported by positive data from an open-label, multinational, single-arm Phase III study in children aged 4 to 11 years, which showed improvements over 12 weeks in two clinically relevant hallmarks of the condition, reduced lymphadenopathy and increased naive B cells, together indicating correction of the underlying immune defect. The submission also includes additional scientific and clinical pharmacology assessments supporting the proposed dosing in lower-weight pediatric patients.
The FDA grants Priority Review to applications for medicines that, if approved, would offer significant improvements in effectiveness or safety of the treatment, prevention, or diagnosis of serious conditions.
“Today’s acceptance and Priority Review of our sNDA marks yet another important step in our efforts to expand access to Joenja for younger children living with APDS. Following the recent approval of Joenja for children aged 4 to 11 years weighing at least 27 kg, this review brings us closer to the possibility of reaching smaller children who currently are ineligible for treatment with Joenja. We look forward to working with the FDA and making Joenja available to eligible pediatric patients as efficiently as possible.” – Anurag Relan, Chief Medical Officer of Pharming.
The FDA approved Joenja for adults and pediatric patients aged 12 years and older with APDS in March 2023 and expanded the approval in September 2026 to include children aged 4 to 11 years weighing at least 27 kg.
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immunodeficiency that was first characterized in 2013. APDS is caused by variants in either one of two identified genes known as PIK3CD or PIK3R1, which are vital to the development and function of immune cells in the body. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immunodeficiency and dysregulation. APDS is characterized by a variety of symptoms, including severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy. Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, it has been reported that people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay. As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma. A definitive diagnosis can be made through genetic testing. APDS affects approximately 1 to 2 people per million worldwide.
