BioPharma, Pharma

J&J, Legend win FDA nod for cell therapy challenger to BMS in multiple myeloma

Approval of the Johnson & Johnson and Legend Biotech cell therapy, Carvykti, marks the second CAR T-cell therapy to clear the regulatory bar for multiple myeloma. The FDA approved Bristol Myers Squibb’s cell therapy Abecma last year.


A cell therapy from Johnson & Johnson and partner Legend Biotech is now FDA approved for treating multiple myeloma, providing a new treatment option for patients with the most serious cases of this type of cancer.

The J&J drug, named Carvykti, is part of a class of personalized, one-time cancer treatments called CAR T, which are made by engineering a patient’s T cells to go after a particular target on the surface of cancer cells. In the case of Carvykti, that target is a protein, B-cell maturation antigen (BCMA), which is found in abundance on the surface of multiple myeloma cells. FDA approval of Carvykti late Monday makes the drug the second approved BCMA-targeting cell therapy for multiple myeloma. Abecma, a CAR T-cell therapy from Bristol Myers Squibb and partner 2Seventybio, was approved by the regulator last year.

Multiple myeloma is a blood cancer that affects plasma cells, a type of white blood cell found in the bone marrow. These cancerous plasma cells spread and replace healthy cells, forming tumors in the bone marrow. It’s a relatively rare cancer, accounting for an estimated 34,920 cases out of about 1.9 million cancer cases diagnosed last year, according to the American Cancer Society.

Though Abecma and Carvykti both go after BCMA, the J&J cell therapy expresses a receptor that has two BCMA-targeting single domain antibodies, which the company says gives it stronger ability to bind to the target cancer protein. The approval of Carvykti covers its use in adults whose multiple myeloma has relapsed or has not responded to four or more earlier treatments, putting it on par with Abecma’s drug label. The FDA based its Carvykti approval decision on data from a pivotal Phase 1b/2 clinical trial. The open-label study enrolled patients who had received at least three prior lines of treatment.

Preliminary data for the first 29 patients were reported at the 2020 annual meeting of the American Society of Clinical Oncology, showing a 100% overall response rate to treatment, and a stringent complete response rate of 76% (22 patients). Updated data for 97 patients were presented at the annual meeting of the American Society of Hematology in December. The results showed an overall response rate of 98% with 83% of patients showing a stringent complete response at a follow-up of a median 22 months.

Carvykti’s drug label carries a boxed warning that cautions clinicians and patients that the drug may cause a dangerous immune response called cytokine release syndrome; neurotoxicity; Parkinsonism; and Guillain-Barré syndrome, which is an immune response that affects the nerves. The drug labels of Abecma and other CAR T-drugs carry similar warnings. Like Abecma, the new J&J cell therapy is available only under a program that informs clinicians and patients about the risks of the treatment.

Approval of Carvykti comes later than initially expected. Last November, J&J announced that the FDA needed more time to review additional information the company had submitted at the agency’s request. That pushed the target date for a decision to Feb. 28.

Carvykti was initially developed by Legend Biotech, a China-based company that maintains U.S. operations in Somerset, New Jersey. In 2017, J&J subsidiary Janssen entered into a global licensing and collaboration agreement with Legend Biotech covering the development and commercialization of the cell therapy. Under the deal, the companies will share in the production and commercialization costs of the cell therapy, as well as the profits for the drug worldwide except for China, which is split 70% to Legend and 30% to Janssen.

Janssen has set a wholesale price of $465,000, which is higher than the $419,500 price that BMS announced for Abecma after its approval. In an emailed statement, Janssen said that the price “reflects the significant clinical value of this cell therapy in addressing an unmet medical need in heavily pretreated patients with limited treatment options. We anticipate Carvykti will be covered by most commercial payers and Medicare as a medical benefit.”

Photo: Niels Wenstedt/BSR Agency, Getty Images