On 24 June 2021, the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product idecabtagene vicleucel (Abecma), intended for the treatment of relapsed and refractory multiple myeloma. As Abecma is an advanced therapy medicinal product, the CHMP’s positive opinion is based on an assessment by the EMA’s Committee for Advanced Therapies.
The applicant for this medicinal product is Celgene Europe BV.
Abecma will be available as a 260-500 x 10⁶ CAR-positive viable T cells dispersion for infusion. The active substance of Abecma is idecabtagene vicleucel, a chimeric antigen receptor (CAR)-positive T cell therapy targeting B-cell maturation antigen (BCMA), which is expressed on the surface of normal and malignant plasma cells. Antigen-specific activation of Abecma results in CAR-positive T cell proliferation, cytokine secretion and subsequent cytolytic killing of BCMA-expressing cells.
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.
The benefits of Abecma are its ability to provide durable responses in patients with relapsed and refractory multiple myeloma.
The main study on which the recommendation for a conditional marketing authorisation is based was a phase II, multicentre, open label, single-arm clinical study. 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 for about 11 months on average. Of those studied, 30% showed complete response.
The most common side effects are neutropenia, cytokine release syndrome, anaemia, thrombocytopenia, unspecified infections, leucopenia, fatigue, diarrhoea, hypokalaemia, hypophosphataemia, nausea, lymphopenia.
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.
The full indication is:
Abecma is indicated for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.
Abecma should be prescribed by physicians experienced in the treatment of haematological malignancies including multiple myeloma.
This product was designated as an orphan medicine on 20 April 2017. Following this positive CHMP opinion, the EMA’s Committee for Orphan Medicinal Products will assess whether the orphan designation should be maintained.
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. Abecma was accepted into the PRIME scheme on 10 November 2017.
Additional efficacy and safety data are being collected through the submission of follow-up data from the main clinical study 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.
Detailed recommendations for the use of this product will be described in the summary of product characteristics, which will be published in the European public assessment report and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.
Summaries of positive opinion are published without prejudice to the Commission decision, which will normally be issued 67 days from adoption of the opinion.
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 EU Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.