Actemra Gets FDA Approval for Cytokine Release Syndrome Treatment

Mathew Shanley

On the same day that the first chimeric antigen receptor (CAR)-T-based gene therapy was approved by the U.S. Food and Drug Administration (FDA), the government agency has also approved a drug to help with a life-threatening side effect associated of it.

Genentech announced that its drug Actemra (toclizumab) has received FDA approval for the treatment of CART cell-induced severe or life-threatening cytokine release syndrome (CRS) in patients 2 years of age and older.

The drug is to be administered via intravenous injection, and was granted Priority Review and Orphan Drug Designation based on the potentially fatal nature of CRS.

CRS is a common immediate complication occurring with the use of anti-T cell antibody infusions. It is an overactive immune response, and has been confirmed as a severe and life-threatening side effect of CAR T cell therapy for certain cancers. Common symptoms are flu-like, including high fevers, which are easily managed, because CRS is essentially a form of non-infective fever.

Some patients, however, experience more serious symptoms that could lead to potentially life-threatening difficulties like cardiac dysfunction, acute respiratory distress syndrome or multi-organ failure.

“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, M.D., chief medical officer and head of Global Product Development in a press release.

“Today's approval of Actemra for cytokine release syndrome provides physicians with an important tool to help manage this potentially life-threatening side effect.”

The impetus for the approval was the successful data from a clinical trial of CAR T cell therapies for blood cancers, which evaluated the efficacy of Actemra in the treatment of CRS. In the study, 45 pediatric and adult patients with severe or life-threatening CRS were treated with Genentech’s therapy, with or without additional high-dose corticosteroids. 31 patients achieved a response with a maximum of 2 doses of Actemra and no adverse reactions were reported.

In a second study involving a cohort of 15 patients, resolution of CRS was confirmed within 14 days of administering Actemra.

Common side effects in patients treated with Actemra included upper respiratory tract infections, headaches, increased blood pressure, and injection site reactions.

Acterma has previously been used for the treatment of adults with rheumatoid arthritis, but is also approved for 2 other rare conditions; polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis.

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