Pharming Group’s Pediatric Trial Yields Positive Results

Pharming Group (PHGUF) has released an update.

Pharming Group announced promising topline results from its Phase III clinical trial of leniolisib, targeting children aged 4 to 11 with activated phosphoinositide 3-kinase delta syndrome (APDS). The trial showed improvements in lymphoproliferation and immunophenotype correction, with no serious adverse events reported, paving the way for global regulatory filings in 2025. This development offers hope for younger patients with this rare immunodeficiency, potentially expanding treatment options for an underserved group.

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