European Medicines Agency recommends CAR T-cell approvals

The European Medicines Agency (EMA) has recommended a handful of hematology medications for approval, including two chimeric antigen receptor (CAR) T-cell therapies.

All of the drugs must next be approved by the European Commission in order to be marketed to patients throughout Europe.

At the end of June, the EMA’s Committee for Medicinal Products for Human Use recommended granting marketing authorization to Europe’s first two CAR T-cell therapies: tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta).

^{Courtesy Novartis}

The EMA approval recommendations come with risk management measures to address the potential for cytokine release syndrome with both of these treatments. Drug makers must use a patient registry to monitor the long-term safety and efficacy of the therapies.

The EMA is also recommending approval of caplacizumab for acquired thrombotic thrombocytopenic purpura, vonicog alfa for the treatment of von Willebrand disease, and daunorubicin/cytarabine for the treatment of acute myeloid leukemia.

mschneider@mdedge.com