Advanced therapy medicinal products (ATMPs) – An analysis of the global regulatory status

BACKGROUND: A rapid progress in the development of advanced therapy medicinal products (ATMPs) has taken place over the past decades. The ATMPs hold great potential but face challenges in the clinical development and the regulatory process. OBJECTIVE: To investigate the current global regulatory status of ATMPs by systematically identify, characterize and compare ATMPs granted marketing authorization (MA) by regulatory agencies across the globe. METHODS: We performed an analysis of ATMPs with MA on January 1, 2025, granted by the regulatory agencies in USA, EU, Japan, UK, Canada, Australia and Switzerland. RESULTS: A total of 59 different ATMPs had been granted an MA by at least one of the included agencies. Gene therapy medicinal product was the most common type of ATMP (42%), and haematology was the most common therapeutic area (44%). The number of ATMPs that had been granted MA ranged from 43 in the USA to 7 in Australia. Five (8%) of the ATMPs had been granted MA across all the included agencies, while 17 (29%) of the ATMPs had been granted MA by more than one agency. CONCLUSIONS: This study showed substantial variation between agencies in the number of ATMPs that had been granted MA. Around one-third of the ATMPs had been granted MA by more than one agency. Assuming that the ATMPs are offering a clinical benefit for the patients, limited global dissemination is a problem as it hinders the accessibility to patients globally resulting in health care inequality.