FDA Approves Ciltacabtagene Autoleucel for Relapsed/Refractory Multiple Myeloma

Ciltacabtagene autoleucel (cilta-cel) has been approved by the US Food and Drug Administration (FDA) for the treatment of patients with relapsed or refractory multiple myeloma (MM) who have received 4 or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

The agency’s decision to approve the B-cell maturation agent (BCMA)–directed chimeric antigen receptor (CAR) T-cell therapy is based on findings from the CARTITUDE-1 trial. The overall response rate for patients in the study with relapsed/refractory MM who had received a median of 6 prior therapies was 98% (n, 97; 95% CI 92.7-99.7). In addition, 78% (n, 76; 95% CI 68.8-86.1) achieved stringent complete response. After a median follow-up of 18 months, the median duration of response was 21.8 months.

Common adverse events (AEs) reported in at least 20% of patients treated with cilta-cel were pyrexia, cytokine release syndrome (CRS), hypogammaglobulinemia, hypotension, musculoskeletal pain, fatigue, infections-pathogens unspecified, cough, chills, diarrhea, nausea, encephalopathy, decreased appetite, upper respiratory tract infection, headache, tachycardia, dizziness, dyspnea, edema, viral infections, coagulopathy, constipation, and vomiting. The prescribing information for the CAR T-cell therapy contains boxed warnings for CRS, immune effector cell-associated neurotoxicity syndrome, Parkinsonism and Guillain-Barre syndrome, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, and prolonged or reccurent cytopenias.

The FDA and European Commission granted orphan drug designation to cilta-cel in 2019 and 2020, respectively. Additionally, in 2019, cilta-cel received breakthrough therapy designation in China. The ongoing CARTITUDE-1 study continues to assess the longer-term efficacy and safety of cilta-cel.

Source: Johnson & Johnson press release, February 28, 2022.