Pharming Group N.V. announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for its supplemental New Drug Application (sNDA) for leniolisib. This application seeks approval for leniolisib in children with Activated Phosphoinositide 3-kinase Delta Syndrome (APDS) aged 4 to 11 years.
If approved, leniolisib would be the first and only treatment indicated for this younger pediatric population with APDS, a rare primary immunodeficiency. The FDA's decision is based on positive data from a multinational Phase III study in this age group.
The Prescription Drug User Fee Act (PDUFA) target action date for the FDA's decision is January 31, 2026. The Priority Review designation indicates that the FDA believes the drug, if approved, would provide a significant improvement in the safety or effectiveness of the treatment of a serious condition.
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