NEW YORK – Legend Biotech announced Thursday that the European Commission granted conditional marketing authorization to Carvykti (ciltacabtagene autoleucel) for patients with relapsed and refractory multiple myeloma.
The approval will allow the Somerset, New Jersey-based company and Janssen to market the autologous CAR T-cell therapy as a treatment for patients in Europe who have received at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody, and have progressed on their most recent therapy.
The EC's approval is based on results from the pivotal Phase Ib/II CARTITUDE-1 trial. Patients in that study had received a median of six prior treatments and were followed for a median of 18 months. With a one-time treatment with Carvykti, 98 percent of patients responded to therapy, and 80 percent experienced a complete response.
European regulators also considered safety data for Carvykti from two open-label trials, MMY2001 and MMY2003. Those studies showed a high rate of adverse events, such as neutropenia in 91 percent of patients and cytokine release syndrome in 88 percent. Patients also experienced hematological abnormalities, fever, fatigue, pain, upper respiratory infection, transaminase elevation, and other adverse events.
Carvykti is engineered to target the B-cell maturation antigen, or BCMA, on the surface of multiple myeloma cells. Legend licensed rights to develop and commercialize Carvykti from Janssen in 2017. The latest regulatory action represents Legend's first EC-approved product.
Autologous CAR T-cell therapies involve reprogramming a patient's own T cells to attack cancer cells and, therefore, administering them requires "extensive training, preparation, and certification," Legend said in a statement. Janssen plans to make Carvykti initially available through a small network of certified treatment centers with the expertise to administer the treatment and then steadily increase availability to other treatment centers across Europe.
The US Food and Drug Administration approved this same Carvykti indication in February and required the sponsors to implement a risk evaluation and management strategy to manage the toxicities associated with therapy.
Legend and Janssen continue to collect long-term efficacy and safety data within the ongoing CARTITUTE-1 trial.