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FDA Approves Carvykti for Earlier Treatment in Multiple Myeloma


The decision comes after the FDA Oncologic Drugs Advisory Committee provided a unanimous vote recommending approval. Carvykti is now available to treat patients after just one prior line of therapy.

Carvykti (ciltacabtagene autoleucel) has gained FDA approval to treat patients with multiple myeloma (MM) earlier. The chimeric antigen receptor (CAR) T-cell therapy from Janssen/Johnson & Johnson will now be available for patients who have received just one prior line of therapy. The company submitted the supplemental biologics license application in June 2023.

The approval comes after the FDA Oncologic Drugs Advisory Committee (ODAC) unanimously voted to recommend Carvykti for approval. ODAC reviews and evaluates the safety and efficacy of drugs at the FDA’s request, but the committee’s recommendations are non-binding.

“The results of the CARTITUDE-4 study demonstrated the substantial clinical benefit of cilta-cel infusion over standard of care continuous therapy in patients who experience a relapse after one to three prior treatments,” Surbhi Sidana, M.D., assistant professor of medicine, Blood and Marrow Transplantation & Cellular Therapy at Stanford University School of Medicine, said in a statement. “This expanded FDA approval for cilta-cel will allow a wider patient population to access this novel therapy earlier in the course of their treatment.”

CAR T-cell therapies have advanced treatment for patients with certain cancers. They offer sustained remission, fewer side effects, and a short treatment duration. However, they come with a hefty price tag. Between the cost of drug acquisition and administration, as well as management of adverse events (AEs), the cost for Carvykti is more than half a million dollars per patient per treatment. In some patients, the treatment might need to be repeated.

The new approval was based on data from CARTITUDE-4, an ongoing phase 3, randomized, open-label trial of 419 patients assigned in a randomized fashion to receive either Carvykti (n = 208) or standard care (n = 211). The results were published in New England Journal of Medicine. The patients had received one to three lines of prior therapy, including a proteasome inhibitor and an immunomodulatory drug.

The study results showed Carvykti reduced the risk of disease progression or death by 74% compared with standard care. At 12 months, the progression-free survival was 75.9% for the group treated with Carvykti compared with 48.6% for the standard-care group. In addition, 73.1% of patients receiving Carvykti had a complete response or better compared with 21.8% of patients on standard care.

An analysis of the phase 3 results presented at the 2023 American Society of Hematology annual meeting also highlighted the clinically meaningful improvements in health-related quality of life. Patients on Carvykti reported improved functioning and symptom reduction from baseline.

Grade 3 or 4 adverse events (AEs) occurred in 96.6% of patients receiving Carvykti and 94.2% of patients on standard care. The most commonly occurring AEs in both groups were hematologic: neutropenia, thrombocytopenia, anemia, and lymphopenia. The standard care group had a higher rate of infections (71.2% any grade) compared with the Carvykti group (62.0%).

Three-fourths (76.1%) of patients on Carvykti experience cytokine release syndrome, while 20.5% of patients experienced neurotoxicity, two CAR T-cell therapy–associated AEs. Fourteen patients in the Carvykti group died from disease progression, although eight had not received the CAR T-cell therapy, compared with 30 patients in the standard of care group.

Carvykti was first approved in February 2022 to treat patients with MM who had been treated with four or more lines of therapy. This new approval, moving the CAR T-cell therapy into a much earlier line of care, is part of a trend for these therapies. In April 2022, Yescarta (axicabtagene ciloleucel), the very first CAR T-cell therapy approved in the United States in 2017, was approved as a second-line treatment for large B-cell lymphoma. There is even research into Yescarta as a first-line treatment for certain high-risk patients.

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