ADVANCED THERAPY MEDICINAL PRODUCTS – AN OVERVIEW

Advanced therapy medicinal products (ATMPs) represent an innovative group of medicines comprising gene therapies, somatic cell therapies and tissue engineered products. These therapies offer opportunities for new therapeutic approaches in numerous diseases. However, there are many issues related to the development of such complex therapies that greatly differ from traditional developments of new chemical entities or biologics. The regulatory framework, high cost of development, translation from research to clinical trials and finally into clinical practice are still issues that make marketing authorization (MA) of ATMPs in the European Union (EU) a very challenging task. Nine years after the implementation of a common regulatory framework for ATMPs in the EU, only 9 ATMPs have been granted MA. Three of them are not available on the EU market anymore. This overview aims to give a short insight into the challenges this innovative field of medicines is facing today.