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EMA positive opinion for new gene therapy to treat multiple myeloma

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EMA has recommended granting a conditional marketing authorisation for Carvykti (ciltacabtagene autoleucel) and it is now pending for European Commission decision.

Medicines for human use are eligible for conditional marketing authorisation if they are intended for treating, preventing or diagnosing seriously debilitating or life-threatening diseases. Regarding this principle, Carvykti is a gene therapy indicated 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

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.

Multiple myeloma is a debilitating and life-threatening disease particularly because it disrupts the normal functioning of the bone marrow, damages the bones, and causes kidney failure. Despite the development and approval of a range of medicines for the treatment of multiple myeloma over the past years, there are limited therapeutic options for patients who have already received three major classes of drugs (immunomodulatory agents, proteasome inhibitors and monoclonal antibodies). Therefore, multiple myeloma is an unmet medical need and new medicines are needed for these patients.

For these reasons, 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 patient’s unmet medical needs. Also, as multiple myeloma affects approximately to 4 in 10.000 people in the European Union, this medicine was also granted Orphan designation in 2020.

Gene therapy

The active substance of Carvykti, ciltacabtagene autoleucel, is a genetically modified autologous T cell immunotherapy consisting of modified T-cells bearing a chimeric antigen receptor (CAR) targeting B-cell maturation antigen (BCMA). BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B cells and plasma cells. Upon binding to BCMA-expressing cells, the CAR promotes T cell activation, expansion and elimination of target cells.

Carvykti is an advanced gene 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.

Clinical trial

The recommendation for a conditional marketing authorisation is based on a single arm, open-label, multicentre clinical trial. This study investigated the efficacy and safety of ciltacabtagene autoleucel in 113 adult patients with relapsed and refractory multiple myeloma who had received at least three prior therapies: immunomodulatory agent, proteasome inhibitor and anti-CD38 antibody.

About 84% of patients enrolled in the study responded to the treatment with a durable response (a period without disease signs or symptoms after treatment). Around 69% showed a complete response, meaning the signs of cancer disappeared.

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, infection, and encephalopathy. Other important safety aspects are neurologic toxicity, prolonged cytopenia and serious infection, so monitoring and mitigation strategies for these side effects are part of the authorisation.

If you want to know more about new gene therapy to treat multiple myeloma, check out the official press release here.

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