FDA Approves Carvykti as Targeted CAR-T Cell Therapy for Multiple Myeloma

The FDA has approved Legend Biotech's Carvykti® as the first BCMA-targeted CAR-T cell therapy for the second-line treatment of multiple myeloma, offering new hope to patients with relapsed or refractory disease.

Legend Biotech announced that the US Food and Drug Administration (FDA) has granted approval to its CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) for the treatment of multiple myeloma. This therapy is the first and only BCMA-targeted CAR-T cell treatment approved for use as early as the second line of defense. This groundbreaking approval positions CARVYKTI® at the forefront of multiple myeloma therapy, ahead of other CAR-T therapies, bispecific antibodies, and antibody-drug conjugates.

“The expanded label of CARVYKTI has the potential to transform the treatment paradigm for multiple myeloma by providing patients and physicians with a personalized immunotherapy that can be used earlier in the treatment regimen. Multiple myeloma is an incurable and progressive hematologic cancer that causes patients to relapse and become refractory to treatment, so new, innovative options are desperately needed,” said Ying Huang, CEO of Legend Biotech.

The FDA's decision was propelled by compelling data from the CARTITUDE-4 study, which underscored CARVYKTI®'s efficacy in significantly enhancing progression-free survival compared to standard care treatments. This unanimous recommendation from the FDA Oncologic Drugs Advisory Committee underlines the therapy's considerable clinical benefit, offering a new lease on life for patients who have exhausted other treatments.

Dr. Surbhi Sidana, from Stanford University School of Medicine, lauded the expanded approval as a milestone that broadens the accessibility of this innovative therapy to a larger group of patients earlier in their treatment regime. This is a critical development, as it addresses the growing need for more effective treatments following the first relapse.

In anticipation of increased demand, Legend Biotech has ramped up its manufacturing and supply chain capabilities. Birk Vanderweeën, Senior Vice President, highlights the company's commitment to ensuring that patients who need CARVYKTI® can access it, marking a significant achievement in personalized healthcare.

The therapy's safety profile includes warnings for Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), among other potential complications. With common adverse reactions ranging from pyrexia and hypotension to more serious conditions such as lymphopenia and neutropenia, the path forward with CARVYKTI® will be navigated with vigilant monitoring and care.