Genetic renaissance: a cross-regional analysis of the approval landscape of authorised gene therapeutics in paediatrics, challenges and future prospects.

Abstract

Gene therapies are proven to be a milestone in the treatment of genetic disorders, especially in children who bear a disproportionately high burden of rare and hereditary diseases. Clinical evaluation of gene therapy (GT) in paediatrics is a significant challenge for every regulatory body. This study examined the available data on GT products that have been authorised for use by children in Japan, Europe, and the United States. We systematically analysed publicly available regulatory databases from USFDA, EMA, and PMDA to track GT approvals. Therapies were categorized based on their intended patient population (adults, paediatrics, or both), approval trends, and regulatory designations such as Orphan Drug Designation (ODD), Fast Track Designation (FTD), Breakthrough Therapy Designation (BTD), and Rare Paediatric Disease Designation (RPDD). As of April 2024, a total of 75 GTs were approved across these three regions for adults, paediatrics, and both populations combined. 37 in the US, 18 in Europe, and 20 in Japan. Among them, 41 were for adults, 13 for paediatrics, and 21 for both age groups. Of the 13 paediatric approvals the USFDA leads in paediatric GT approvals with 7 therapies, followed by the EMA with 5 (of which 2 were later withdrawn due to commercial reasons), and the PMDA with 1 therapy. Gene therapies hold immense promise for paediatric patients, offering life-changing treatments where few or no options exist. However, high costs, complex clinical trial requirements, and long-term safety concerns continue to limit their widespread adoption. This study underscores the urgent need for global regulatory harmonization and policy initiatives to improve access to paediatric GTs. While regulatory frameworks have enabled faster approvals, sustained efforts are required to ensure affordability, long-term safety, and equitable access for children worldwide.