• Actemra/RoActemra® is the first FDA approved drug for treatment of serious or fatal cytokine release syndrome (CRS) caused by CAR-T therapy;
  • CAR-T cell therapy is an immunotherapy used to treat certain types of cancer;
  • This is its seventh approval by FDA since Actemra® has been listed in the United States from 2010.

Roche Group announced this month that the U.S. Food and Drug Administration (FDA) had approved Actemra/RoActemra (tocilizumab) intravenous injection for the treatment of severe or life-threatening cytokine release syndrome (CRS) in children over age 2 and adults caused by chimeric antigen receptor (CAR) T cell therapy. CRS, produced by excessive immune response, is a severe and life-threatening side effect in the CAR-T therapy for the treatment of certain types of cancer.

Dr. Sandra Horning, the Roche’s chief medical officer and head of global product development, said,

“Previously, there had been no FDA approved drug that can be used to treat severe cytokine release syndrome caused by CAR-T cell therapy. This disease rapidly occurs and can lead to life-threatening complications.

Now, the FDA’s approved of Actemra® for the treatment of cytokine release syndrome provides an important boost for clinicians, helping them to control this life-threatening side effects.”

The approval is based on a retrospective analysis of the clinical data from CAR-T cell therapy on leukemia, to assess therapeutic effect of Actemra® on cytokine release syndrome. And study group comes from 45 children and adult patients with severe or life-threatening cytokine release syndrome. By whether accepting extra high doses of corticosteroids treatment or not, these patients are divided into two groups in the process of Actemra® treatment.

31% of the patients (69%; 95% CI: 53%-82%) achieved evaluation standard (eliminate cytokine release syndrome) 14 days after the first injection of Actemra®, without injection of second Actemra®, and without the use of any drugs except for Actemra® and corticosteroids. Besides, any Actemra®related adverse reactions had not been found during the treatment. The second research by using only Actemra® showed that 15 patients, having cytokine release syndrome due to the CAR-T therapy, were cured 14 days later by using Actemra® treatment. It confirms that Actemra® does have treatment effect on such symptom.

Based on the rare and serious characteristics of cytokine release syndrome and Actemra®’ safety and efficacy data, FDA grants Actemra® priority review and rare disease drug qualification for the treatment of CRS caused by CAR-T cell therapy. The drug received priority review specified qualification is that it can greatly improve the safety and efficacy in treatment of serious illness. Drugs that are qualified for rare disease drug qualification are used primarilyto treat diseases fewer than 200,000 in the United States.CAR-T Therapy Drug

About cytokine release syndrome caused by CAR-T cell therapy

CAR-T cell therapy aims to change the cells of individual patients to target cancer cells, and to treat certain types of leukemia. Cytokine release syndrome, caused by excessive immune reaction, has been regarded as a potentially serious and life-threatening side effect in the CAR-T cell therapy.

Most cytokines release syndrome patients will appear mild or moderate flu like symptoms, and these symptoms are easily managed. However, some patients may exhibit more severe symptoms which may cause life-threatening complications, such as cardiac dysfunction, acute respiratory distress syndrome or multiple organ failure.

Editor’s note:

When Novartis, Juno and cellectis are struggling in the fighting of being the first company who has FDA approval of CAR-T therapy, Roch has already get FDA approval for its antibody drug for the treatment of Cytokine release syndrom caused by CAR-T therapy. Obviously, Roch is profiting from this business war without any risks. I don’t know how their faces will look like when the senior managements of the three bio-pharma giants hear this news. I just want to say, well done, Roch, well done!