Legend Biotech Corp., a global biotechnology company developing novel therapies to treat life-threatening diseases, received its first approval from the U.S. FDA for CARVYKTI (ciltacabtagene autoleucel; cilta- cel), for the treatment of adults with relapsed or refractory multiple myeloma (RRMM) who have received four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. Legend Biotech entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. to develop and commercialize cilta- cel in December 2017. 
 

CARVYKTI is a chimeric antigen receptor T-cell (CAR-T) therapy with two B-cell maturation antigen (BCMA)-targeting single domain antibodies and given as a one-time infusion. In the pivotal CARTITUDE-1 study, deep and durable responses were seen in patients with RRMM (n=97), with a high overall response rate (ORR) of 98 percent, including 78 percent of the patients achieving stringent complete response. At a median of 18 months follow-up, the median duration of response (DOR) was 21.8 months (95 percent).
 

CARVYKTI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the CARVYKTITM REMS Program.1 The Safety Information for CARVYKTI includes a Boxed Warning regarding Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), Parkinsonism and Guillain-Barré syndrome, hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), and prolonged and/or recurrent cytopenia. The most common adverse reactions (≥20 percent) are pyrexia, CRS, hypogammaglobulinemia, hypotension, musculoskeletal pain, fatigue, infections-pathogen unspecified, cough, chills, diarrhea, nausea, encephalopathy, decreased appetite, upper respiratory tract infection, headache, tachycardia, dizziness, dyspnea, edema, viral infections, coagulopathy, constipation, and vomiting.
 

Multiple myeloma affects a type of white blood cell called plasma cells, which are found in the bone marrow.2 The majority of patients relapse after undergoing initial treatment and face poor prognoses after treatment with three major drug classes, including immunomodulatory agent, a proteasome inhibitor and anti-CD38 monoclonal antibody.