FDA Approves Carvykti, a CAR-T Cell Therapy, for Pretreated Relapsed/Refractory Myeloma

Ninety-eight percent of patients with pretreated relapsed/refractory myeloma responded to treatment with Carvykti, a newly approved CAR-T cell therapy for this patient population.

The Food and Drug Administration (FDA) approved the CAR-T cell therapy, Carvykti (cilta-cel), for the treatment of adults with relapsed/refractory multiple myeloma who underwent four or more previous treatments, according to Janssen and Legend Biotechs, the manufacturers of the therapy.

The previous treatments in this patient population include a proteasome inhibitor, an immunomodulary agent and an anti-CD38 monoclonal antibody, according to the manufacturers.

The FDA approval is based on findings from the CARTITUDE-1 clinical trial, which showed that 98% of patients with pretreated relapsed/refractory myeloma responded to treatment with Carvykti. After only one treatment with the CAR-T cell therapy, 78% of patients achieved a stringent complete response, which is when clinicians are unable to detect any signs of disease. At an average follow-up of 18 months, the median duration of response was 21.8 months.

After two years of follow-up, 61% of patients did not experience disease progression, which is particularly exciting, considering that with current standard of care, the average survival for this patient population is about nine months.

“The treatment journey for the majority of patients living with multiple myeloma is a relentless cycle of remission and relapse with fewer patients achieving a deep response as they progress through later lines of therapy,” said Dr. Sundar Jagannath, a professor of medicine at Mount Sinai in New York and principal investigator of the CARTITUDE-1 trial, in the release from Legend Biotech.

"We are committed to harnessing our science, deep disease understanding and capabilities to bring forward cell therapies like Carvykti as we continue to focus on our ultimate goal of delivering a cure for multiple myeloma," said Dr. Peter Lebowitz, Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC, in a statement.

Carvykti is available through a restricted program called the Carvykti Risk Evaluation and Mitigation Strategy (Carvykti REMS), meaning that patients must receive the CAR-T cell therapy in a center where clinicians are trained to administer the treatment.

The drug included a boxed warning regarding cytokine release syndrome (which may lead to fever, nausea, rash, headache and rapid heartbeat, among other symptoms), immune effector cell-associated neurotoxicity syndrome (a somewhat common side effect of CAR-T cell therapy), Parkinson’s disease and Guillain-Barré syndrome, hemophagocytic lymphohistiocytosis (an aggressive, life-threatening syndrome when the immune system is excessively activated) or macrophage activation syndrome (another condition related to excessive activation of the immune system), and prolonged and/or recurrent cytopenias (a condition associated with lower than normal levels of blood cells). Patients should contact their health care providers immediately if they experience any symptoms — including confusion, nausea or headache — after receiving Carvykti.

Common side effects from the drug that occurred in at least 20% of patients given Carvykti on CARTITUDE-1 were fever, cytokine release syndrome, an inability to make antibodies called immunoglobulins, low blood pressure, musculoskeletal pain, fatigue, infections of unspecific pathogens, cough, chills, diarrhea, nausea, brain toxicity, decreased appetite, upper respiratory tract infection, headache, fast heartbeat, dizziness, shortness of breath, swelling, viral infections, excessive bleeding or clotting, constipation and vomiting.

"We extend our sincere gratitude to the patients, their families and the teams of researchers and study centers who have participated in the clinical study of Carvykti and enabled today's approval,” Lebowitz said.

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