EMA Grants Recommendation for New Gene Therapy to Treat Adult Patients with Multiple Myeloma

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EMA has recommended a conditional marketing authorization for a new gene therapy to treat adult patients with multiple myeloma.

EMA has recommended a conditional marketing authorization in the 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 that affects the plasma cells, a type of white blood cell found in bone marrow that produces antibodies. Plasma cells uncontrollably increase in numbers, resulting in abnormal, immature plasma cells multiplying and filling up the bone marrow. Cancerous plasma cells no longer protect the body from infections and instead produce abnormal proteins that can cause problems in the kidneys, bones, or blood.

There are currently limited therapeutic options for patients whose disease has returned or no longer responds to existing medicines, which is why new medicines are needed for these patients.


Ciltacabtagene autoleucel is a chimeric antigen receptor (CAR)-T cell medicine that collects and modifies a patient’s own immune T-cells to create a personalized treatment that is then infused back into the patient. The recommendation will now be sent to the European Commission for a decision on the adoption of an EU-wide marketing authorization.

Source: EMA