First two CAR-T cell medicines recommended for approval in the European Union

The European Medicines Agency (EMA) has recommended the first two marketing authorisations for chimeric antigen receptors (CAR) T-cells medicines in the European Union (EU). Both of them, are advanced therapies for blood cancer. They belong to a new generation of personalised cancer immunotherapies that are based on collecting and modifying patients’ own immune cells to treat their cancer.

Both advance therapies, are also the first medicines supported through EMA’s PRIority MEdicines (PRIME) scheme to receive positive opinions from the Committee for Medicinal Products for Human Use (CHMP). The voluntary scheme provides early and enhanced scientific and regulatory support to medicines that have the potential to address, to a significant extent, patients’ unmet medical needs.

“CAR-T cells transform the fight against serious and often fatal diseases in the EU,” said Dr Martina Schüssler-Lenz, chair of the Committee for Advanced Therapies (CAT). “Those two advance therapies medicines, offer an innovative approach where patients’ cells are reprogrammed and reinjected to attack the cancer.”

“Innovative treatments such as CAR-T cells have potential to change the outlook for patients with cancer, but they also come with new scientific and regulatory challenges,” commented Dr Tomas Salmonson, Chair of the CHMP.

“From the beginning, we have worked to establish a robust system of data collection for the post-authorisation phase that would suit the specificities of these two medicines. We have used a wide range of tools – scientific advice, a specific workshop on patient registries for CAR-T cells, PRIME, to name just a few, to enable us to define the methods to tightly monitor the benefit-risk profile of these medicines and manage their risks once they are on the market, so that patients can benefit from these innovative treatments.”