With many in biotech and pharma in China concerned about the relevance of international clinical data to the FDA, there’s additional confirmation of its value with the FDA’s recent approval of Carvykti/Cilta-cel.
Carvykti is a CAR-T immunotherapy for the treatment of relapsed or refractory multiple myeloma (RRMM) in adults from the China-focused Legend Biotech. This is the first approved therapy to come from Legend Biotech, which was spun off from GenScript Biotech. Carvykti/Cilta-cel’s approval first underwent clinical testing in China, followed by further testing in Japan and the US. This comes in the wake of the Sintilimab failure, which relied entirely on data from testing in China, and will require an additional trial in the US for reconsidered approval by the FDA’s Oncologic Drug Advisory Committee. Additionally, Carvykti/Cilta-cel received Orphan Drug Designation from the FDA, as well as Breakthrough Therapy Designation.
Carvykti/Cilta-cel’s approval shows that international clinical data is still valuable, useful, and relevant to seeking approval from the FDA in the US, especially if addressing an unmet need.
Dr. Sundar Jagannath, MBBS, Professor of Medicine, Hematology, and Medical Oncology at Mount Sinai Hospital in New York City and Principal Study Investigator, said that “[The] approval of Carvykti helps address a great unmet need for [RRMM] patients.” Dr. Jagannath continues, “The treatment journey for the majority of patients living with multiple myeloma is a relentless cycle of remission and relapse with few patients achieving a deep response as they progress through later lines of therapy […] Cilta-cel can provide deep and durable responses and long-term treatment-free intervals, even in this heavily pretreated multiple myeloma patient population.”
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Carvykti/Cilta-cel is a CAR-T therapy with B-cell maturation antigen (BCMA) targeting domains, which will reprogram a patient’s own T-cells to identify and destroy cells that express BCMA on their surface. BCMA is expressed primarily on the surface of malignant multiple myeloma B-lineage cells, which when bound to the chimeric antigen receptor (CAR) protein of Carvykti/Cilta-cel results in T-cell activation, expansion, and elimination of the BCMA-presenting cells.
Carvykti/Cilta-cel will be made available exclusively through a Risk Evaluation and Mitigation Strategy (REMS), a restricted program under the FDA for medications that have serious safety concerns to help ensure that the benefits of Carvykti/Cilta-cel outweigh the risks. Risks specifically associated with Carvykti/Cilta-cel include Cytokine Release Syndrome, Immune lymphohistiocytosis/macrophage activation syndrome and prolonged and/or recurrent cytopenia.
However, in collaboration with Janssen Biotech Inc, a subsidiary of Johnson & Johnson, with whom Legend Biotech is jointly developing and commercializing Carvykti/Cilta-cel, there is hope that this new immunotherapy will provide a new option for patients of RRMM. Said Ying Huang, PHD, CEO and CFO of Legend Biotech, “[The] approval of Carvykti is a pivotal moment for Legend Biotech because it is our first-ever marketing approval, but what really excites us the drug’s potential to become an impactful therapy option for patients in need of long, treatment-free intervals.”
Representing the newest wave in personalized medicine, Carvykti/Cilta-cel will require significant preparation, training, and certification to provide the best experience possible for patients. Legend and Janssen will increasingly scale availability of Carvykti/Cilta-cel across the US and beyond in 2022 so that as many oncologists and patients can gain reliable access to the new therapy as possible.
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