Pharming Group N.V. announced positive topline results from its multinational Phase III study evaluating leniolisib tablets in children aged 4 to 11 years with Activated Phosphoinositide 3-kinase Delta Syndrome (APDS). The data demonstrated improvements in lymphoproliferation and immunophenotype correction.
The study enrolled 21 children with APDS, all of whom completed the 12-week treatment period. Improvements were measured by a mean reduction in index lymph node size and an increase in the percent of naïve B cells, consistent with previously reported data in adolescent and adult patients.
All treatment-emergent adverse events were reported as mild to moderate, with no drug-related serious adverse events. Pharming plans to include this data in global regulatory filings for the approval of leniolisib for pediatric patients in this age group, with filings expected to begin in 2025.
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