Pharming announces positive topline data in pediatric clinical trial of leniolisib

Multinational Phase III study is evaluating leniolisib tablets in children aged 4 to 11 years with APDS, a rare primary immunodeficiency Data consistent with the improvements seen in the previously reported randomized controlled trial in adolescent and adult APDS patients Global regulatory filings planned to begin in 2025 Leniolisib, marketed under the brand name Joenja® in the U.S., received approval from the U.S. Food and Drug Administration (FDA) for the treatment of APDS in adult and pediatric patients 12 years of age and older in March 2023. Pharming plans to include data from this 4-11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025. “This is the first data from a clinical trial for younger pediatric patients with APDS, who have a significant unmet need for a disease modifying treatment. We look forward to initiating regulatory filings for these younger pediatric patients in 2025.” — Anurag Relan, MD, MPH, Chief Medical Officer of Pharming Resources For further information, please contact: Media Relations Investor Relations Quick links

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