Janssen, Legend CAR-T therapy CARVYKTI gets FDA nod

The US Food and Drug Administration (FDA) has approved a new cell-based therapy for blood cancer, developed by Janssen and China’s Legend Biotech to treat multiple myeloma.

CARVYKTI (ciltacabtagene autoleucel), formerly known as cilta-cel, is chimeric antigen receptor T-cell (CAR-T) therapy indicated for the treatment of adults with relapsed or refractory multiple myeloma who have received four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. 

As a personalised medicine, CARVYKTI treatment requires extensive training, preparation, and certification to ensure a positive experience for patients. The therapy works by reprogramming a patient’s T-cells with a transgene encoding a CAR that identifies and eliminates cells that express the B-cell maturation antigen.

The Janssen-Legend therapy was initially tested in China before expanding to the US and Japan. Following the FDA decision, CARVYKTI is now Legend’s first product to be approved for use in the US.

CARVYKTI’s approval was based upon results from the ongoing phase1b/2 CARTITUDE-1 study. Of the 97 patients enrolled in the open-label, multi-centre study evaluating ciltacabtagene autoleucel for the treatment of patients with relapsed or refractory multiple myeloma, 99% were refractory to the last line of treatment and 88% were triple-class refractory, meaning their cancer did not respond, or no longer responds, to an IMiD, a PI and an anti-CD38 monoclonal antibody.

Initially, the FDA’s decision was expected to arrive by the end of November, six months after the therapy was granted priority review. However, the regulator requested a three-month extension to allow sufficient time to review information submitted by Janssen.

CARVYKTI is the second CAR-T therapy approved by the FDA in the last 12 months. In March 2021, the regulator greenlit the use of the Abecma, a CAR-T treatment for multiple myeloma developed by Bristol Myers Squibb and 2Seventy bio. 

“Multiple myeloma remains an incurable disease with heavily pre-treated patients facing poor prognoses with limited treatment options,” said Ying Huang, PhD, CEO and CFO of Legend Biotech.

“Today’s approval of CARVYKTI is a pivotal moment for Legend Biotech because it is our first-ever marketing approval, but what really excites us is the drug’s potential to become an impactful therapy option for patients in need of long, treatment-free intervals. This is the first of many cell therapies we plan to bring to patients as we continue advancing our pipeline across disease states.”

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