EMA has recommended a conditional marketing authorisation in the European Union (EU) for Carvykti (ciltacabtagene autoleucel) for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies and whose cancer has worsened since they received their last treatment.

Multiple myeloma is a rare cancer of the plasma cells, a type of white blood cell that produces antibodies and is found in the bone marrow. In multiple myeloma, the proliferation of plasma cells is out of control, resulting in abnormal, immature plasma cells multiplying and filling up the bone marrow. When plasma cells become cancerous, they no longer protect the body from infections and produce abnormal proteins that can cause problems affecting the kidneys, bones or blood.

Despite the development and approval of a range of new medicines for the treatment of multiple myeloma over the past few years, there are limited therapeutic options for patients who have already received three major classes of drugs (immunomodulatory agents, proteasome inhibitors and monoclonal antibodies) and whose disease has come back or no longer responds to these medicines. Therefore, new medicines are needed for these patients.

Ciltacabtagene autoleucel, the active substance of Carvykti, is a chimeric antigen receptor (CAR)-T cell medicine. It is an advanced therapy for cancer that is based on collecting and modifying patient’s own immune T-cells to create a patient personalised treatment that is infused back.

Carvykti was supported through EMA’s PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients’ unmet medical needs.

The most common side effects are cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, infections and encephalopathy, i.e. a brain disorder. The consequences of CRS can be life-threatening and, in some cases, even fatal. Furthermore, other important safety aspects are neurologic toxicity, prolonged cytopenia and serious infections. Monitoring and mitigation strategies for these side effects are described in the product information and in the risk management plan that is an integral part of the authorisation.

Additional risk minimisation measures required from the marketing authorisation holder will ensure that centres that dispense the therapy are qualified to recognise and manage CRS and neurotoxicity associated with the treatment of Carvykti.

Because Carvykti is an advanced-therapy medicinal product (ATMP), it was assessed by the Committee for Advanced Therapies (CAT), EMA’s expert committee for cell- and gene-based medicines, and EMA’s human medicines committee (CHMP), which recommended approval based on the CAT assessment.