European Biotech Pharming Group Touts Positive Data From Pivotal Pediatric Study Of FDA-Approved Immune System Disorder Drug

Core Insights - Pharming Group N.V. announced topline results from a Phase 3 trial of leniolisib for children aged 4 to 11 with activated phosphoinositide 3-kinase delta syndrome (APDS), a genetic disorder affecting the immune system [1][2] - Leniolisib, marketed as Joenja in the U.S., received FDA approval for APDS in patients aged 12 and older in March 2023, with plans to seek approval for younger patients starting in 2025 [2] Study Details - The Phase 3 study enrolled 21 children aged 4 to 11 years, focusing on primary efficacy endpoints such as reduction in lymph node size and increased proportion of naïve B cells after 12 weeks [3][5] - Secondary endpoints included evaluating the impact of leniolisib on health-related quality of life [4] Results - All 21 patients completed the 12-week treatment, showing improvement in lymphoproliferation and immunophenotype correction, consistent with previous results in older patients [5][6] - Treatment-emergent adverse events were mild to moderate, with no serious drug-related adverse events reported [6] Market Reaction - Following the announcement, PHAR stock increased by 11.6%, reaching $9.75 [7]