EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for  Abecma (idecabtagene vicleucel) for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three previous therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and whose cancer has worsened since receiving the last treatment.

Multiple myeloma is a rare cancer of a type of white blood cell called plasma cells. Normal plasma cells are found in the bone marrow and are an important part of the immune system. Plasma cells make the antibodies that enable the body to recognise and attack germs such as viruses or bacteria. 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 no longer respond to these medicines. Therefore, new medicines are needed for patients whose disease returns after treatment.

Abecma is a genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy and the first cell-based gene therapy to treat adult patients with multiple myeloma. Each dose of Abecma is created by collecting a patient’s own T-cells (i.e. white blood cells that help the body fight infections) and genetically modifying them so that they include a new gene that helps the body target and kill the myeloma cells. These modified immune cells are then infused back into the patient’s blood.

Abecma 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 main study on which the recommendation for a conditional marketing authorisation is based was a Phase 2, multicentre, open label, single-arm clinical trial. The study investigated the efficacy and safety of Abecma in 140 adult patients with relapsed or refractory multiple myeloma who had received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and who didn’t respond to the last treatment regimen. About 67% of patients enrolled in the study responded to the treatment and maintained remission (a period without disease signs or symptoms after treatment) for about 11 months on average. Of those studied, 30% showed complete response (i.e. disappearance of signs of cancer).

The main safety concerns related to the administration of Abecma are cytokine release syndrome (CRS) (i.e. a condition causing fever, vomiting, shortness of breath, headache and low blood pressure), neurological toxicity, cytopenias (i.e. low number of cells in the blood) and infections, which can be life-threatening.

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 efficacy and safety data are being collected through the submission of follow-up data from the main clinical trial and through an ongoing study that will compare the efficacy and safety of the medicine with standard triplet regimens in patients with relapsed and refractory multiple myeloma.

Because Abecma 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’s assessment.

The opinion adopted by the CHMP is an intermediary step on Abecma’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.

Notes:

  1. The applicant for Abecma is Celgene Europe BV.
  2. Abecma was accepted into the PRIME scheme on 10 November 2017.
  3. Abecma was designated as an orphan medicinal product on 20 April 2017.
  4. Following this positive CHMP opinion, the Committee for Orphan Medicinal Products (COMP) will assess whether the orphan designation should be maintained.

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