Europe – Progress update on pilot for academic and non-profit developers of advanced therapy medicines

Following the launch in September 2022, EMA has accepted three academic and non-profit organisations developing advanced therapy medicinal products (ATMPs) into a pilot scheme, in which they benefit from enhanced support from the Agency.

The aim is to guide non-commercial developers of promising ATMPs addressing unmet medical needs through the regulatory and scientific requirements in the European Union (EU) and better understand their needs to enable them to advance the development of their medicines and eventually reach the marketing authorisation application stage. EMA is looking to add another two developers by the end of 2024.

The first participant selected to join at the start of the pilot is the Hospital Clínic de Barcelona which is developing ARI-0001, a chimeric antigen receptor (CAR) product based on patients’ own T-cells. The medicine is intended for the treatment of patients older than 25 years with relapsed/refractory acute lymphoblastic leukaemia, a type of blood cancer. In December 2021, it was granted eligibility to PRIME, EMA’s scheme to support the development of medicines that target an unmet medical need…