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The U.S. Food and Drug Administration (FDA) has accepted Pharming Group’s supplemental New Drug Application (sNDA) resubmission for Joenja (leniolisib), a treatment intended for children aged 4 to 11 diagnosed with activated phosphoinositide 3-kinase delta syndrome (APDS). This regulatory milestone moves the drug closer to a potential label expansion, as the agency now begins its formal review process to determine if the medication meets safety and efficacy standards for this younger patient population.
The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of September 28, 2026, to complete its evaluation of the resubmission. Joenja, an oral small-molecule inhibitor of the phosphoinositide 3-kinase delta (PI3Kδ) enzyme, currently holds FDA approval for the treatment of APDS in adults and adolescents aged 12 years and older. The resubmission includes clinical data intended to support the use of the therapy in the 4-to-11 age group, addressing the specific medical needs of children living with this rare primary immunodeficiency. The company expects the FDA to conduct a standard review of the provided data before issuing a final decision on the expanded indication.
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Date: June 4, 2026
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