This article has been updated to include Carvykti's list price.
NEW YORK – The US Food and Drug Administration on Monday approved Janssen Pharmaceuticals and Legend Biotech's autologous CAR T-cell therapy Carvykti (ciltacabtagene autoleucel) for previously treated, relapsed, or refractory multiple myeloma patients.
To be eligible for treatment with Carvykti, patients must have received at least four lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
The FDA approved Carvykti based on results from the Phase Ib/II CARTITUDE-1 study, in which 98 percent of patients responded to the one-time treatment, and 78 percent of responders had a complete response. At a median follow-up of 18 months, the median duration of response was 21.8 months.
Carvykti is manufactured from patients' own harvested immune cells and uses an engineered CAR protein with two single-domain antibodies to target the B-cell maturation antigen, or BCMA, on the surface of multiple myeloma cells.
Janssen and Legend inked a licensing agreement in 2017 to jointly develop and commercialize the therapy and submitted their biologics license application with the FDA in December 2020.
The companies are taking a phased approach to rolling out Carvykti. In a statement, they said that they will offer the treatment first at a limited network of certified treatment centers throughout the US and scale up production capacity to ensure that they can "provide Carvykti treatment to oncologists and their patients in a reliable and timely manner."
According to Janssen and Legend, their drug will be available under a Risk Evaluation and Mitigation Strategy, called the CARVYKTI REMS Program, to manage the risk of immune-related toxicities associated with treatment, such as cytokine release syndrome. The firms will continue to assess the treatment's longer-term efficacy and safety within the ongoing CARTITUDE-1 study. Carvykti's wholesale acquisition cost will be $465,000 for the one-time infusion, according to Janssen.
The companies are also working on making Carvykti available outside the US. In April 2021, Janssen applied for the drug's approval with the European Medicines Agency. The European Commission has granted the therapy orphan drug status and regulators in China have deemed it a breakthrough therapy.
The FDA approval for Carvykti comes roughly a year after the agency approved Bristol Myers Squibb's BCMA-directed CAR T-cell therapy Abecma (idecabtagene vicleucel) for relapsed or refractory multiple myeloma patients, making it the first CAR T-cell therapy to enter the market for this cancer type.