FDA Approves Cell Based Multiple Myeloma Therapy Discovered In China
FDA Approves Cell Based Multiple Myeloma Therapy Discovered In China
Recently, Legend Biotech (NASDAQ: LEGN) announced in Somerset, New Jersey, that the U.S. Food and Drug Administration (FDA) has approved CARVYKTI® (Ciltacabtagene Autoleucel, cilta-cel) for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least one prior line of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD), and are refractory to lenalidomide1. CARVYKTI® is the first and only approved B-cell maturation antigen (BCMA)-directed therapy for the treatment of multiple myeloma patients in the second-line setting, including CAR-T therapies, bispecific antibodies, and antibody-drug conjugates (ADCs). Ciltacabtagene Autoleucel also becomes the world’s first and only approved CAR-T product for second-line treatment of multiple myeloma.
Ciltacabtagene Autoleucel is the flagship product of the Chinese company Legend Biotech. This drug was first approved for marketing in the United States on February 28, 2022, for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. It is the first China-originated CAR-T cell therapy drug to be approved outside of China. This drug is the seventh CAR-T product to be approved globally and the second BCMA-targeted CAR-T product. Following its approval in the U.S., Ciltacabtagene Autoleucel has also been approved for marketing in the European Union and Japan. The drug is currently undergoing a marketing application in China and has entered the priority review stage.
The FDA’s approval is based on the positive results of the CARTITUDE-4 study, which showed that compared to two standard treatment regimens, Pomalidomide, Bortezomib, and Dexamethasone (PVd) or Daratumumab, Pomalidomide, and Dexamethasone (DPd), CARVYKTI® significantly improved progression-free survival (PFS) in adult patients with relapsed and lenalidomide-refractory multiple myeloma who had received one to three prior lines of therapy. This result was statistically and clinically significant. This approval came after the Oncologic Drugs Advisory Committee (ODAC) unanimously recommended (11-0) supporting CARVYKTI® for front-line treatment.
Industry experts have pointed out that the use of CAR-T therapy as a front-line treatment for cancer not only means that more patients can receive better treatment options at an earlier stage but also represents an important avenue for pharmaceutical companies to achieve revenue growth. Legend Biotech’s 2023 annual report showed that, driven by its outstanding efficacy and safety, CARVYKTI® generated sales revenue of $469 million in the U.S. region in 2023, accounting for 94% of its total revenue.
Overall, the approval of CARVYKTI® for the second-line treatment of MM represents a milestone event for Legend Biotech and even the entire CAR-T industry.