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FDA Approves Janssen, Legend Biotech's Carvykti in Early-Line Multiple Myeloma

NEW YORK – The US Food and Drug Administration on Friday approved Janssen Biotech and Legend Biotech's BCMA-directed CAR T-cell therapy Carvykti (ciltacabtagene autoleuecel) as an early-line therapy for relapsed or refractory multiple myeloma.

The approval is based on results from the Phase III CARTITUDE-4 trial in which median progression-free survival had not been reached in Carvykti-treated patients at 16 months follow-up compared with a median progression-free survival of 12 months for those on standard-of-care therapies including Bristol Myers Squibb's Pomalyst (pomalidomide) with bortezomib and dexamethasone or Janssen's Darzalex (daratumumab) with Pomalyst and dexamethasone. After a year, 76 percent of patients in the Carvykti arm were still alive versus 49 percent in the control arm. The overall response rate was 85 percent in patients treated with Carvykti and 67 percent in those on standard therapy.

To be eligible for the trial, patients must have received one to three prior lines of therapy including a proteosome inhibitor and an immunomodulatory drug and be refractory to Bristol Myers Squibb's Revlimid (lenalidomide). Patients who had previously received a CAR T-cell therapy or any BCMA-targeted therapy were excluded.

The FDA previously approved Carvykti in 2022 as a treatment for multiple myeloma patients who have received at least four lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. However, only about 15 percent of multiple myeloma patients survive to receive a fifth-line treatment. With this latest approval, Janssen, a subsidiary of Johnson & Johnson, will be able to market Carvykti as an option for advanced multiple myeloma patients as early as the first relapse.

In March, the FDA's Oncologic Drugs Advisory Committee voted 11 to 0 in favor of approving Carvykti in this early-line setting, despite a higher rate of early deaths in the cell therapy arm. Members of the committee noted that the single-infusion cell therapy gives patients valuable time "off treatment" between the single infusion and a subsequent recurrence, and they also highlighted the need for better strategies for bridging patients from their prior treatment to Carvykti to minimize progression during the transition.

Tyrone Brewer, president of J&J's Innovative Medicine hematology division in the US, said in a statement that the company has more than doubled its manufacturing capacity for Carvykti and expects to double it again in 2024, in order to meet patient demand following this new approval. "We will continue to invest in our capacity so we can provide this critical therapy to as many patients as possible," Brewer said.

The FDA also on Friday approved BMS and 2seventy bio's CAR T-cell therapy Abecma (idecabtagene vicleucel) for patients with relapsed or refractory multiple myeloma who have previously received two or more lines of treatment. Abecma is already approved for earlier-line use in multiple myeloma in Japan, Switzerland, and the European Union.