Roche announced  that the US Food and Drug Administration (FDA) has approved Actemra/RoActemra (tocilizumab) intravenous injection for the treatment of chimeric antigen receptor (CAR) T cell-induced severe or life-threatening cytokine release syndrome (CRS) in patients two years of age and older. CRS, which is caused by an overactive immune response, has been identified as a potentially severe and life-threatening side effect of CAR T cell therapy for certain cancers.

“Until today, there has never been an FDA-approved treatment to manage severe cytokine release syndrome associated with CAR T cell therapy, which is marked by a rapid onset and can cause life-threatening complications,” said Sandra Horning, MD, chief medical officer and head of Global Product Development. “Today's approval of Actemra/RoActemra for CRS provides physicians with an important tool to help manage this potentially life-threatening side effect.”

The approval is based on a retrospective analysis of pooled outcome data from clinical trials of CAR T cell therapies for blood cancers, which assessed the efficacy of Actemra/RoActemra in the treatment of CRS.2  The study population included 45 pediatric and adult patients treated with Actemra/RoActemra, with or without additional high-dose corticosteroids, for severe or life-threatening CRS. Thirty-one patients (69%; 95% CI: 53%–82%) achieved a response, defined as resolution of CRS within 14 days of the first dose of Actemra/RoActemra, no more than two doses of Actemra/RoActemra were needed, and no drugs other than Actemra/RoActemra and corticosteroids were used for treatment. No adverse reactions related to Actemra/RoActemra were reported.2  A second study confirmed resolution of CRS within 14 days using an independent cohort that included 15 patients with CAR T cell-induced CRS.  

The FDA granted Priority Review and Orphan Drug Designation to Actemra/RoActemra for the treatment of CAR T cell-induced CRS based on the rare, severe and life-threatening nature of CRS and available data on the safety and efficacy of Actemra/RoActemra. Priority Review Designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the safety and effectiveness of the treatment of a serious condition. Orphan Drug Designation may be granted to medicines intended for the treatment of conditions that affect fewer than 200,000 people in the United States.