FDA Makes Historic Approval of Novartis Gene Therapy

Aug 30, 2017

The U.S. Food and Drug Administration approved the first gene therapy available in the United States, offering a new approach to the treatment of cancer and other life-threatening diseases. It approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, M.D. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”

Kymriah, a cell-based gene therapy, is approved for the treatment of patients up to 25 years old with B-cell precursor ALL that is refractory or in second or later relapse. Kymriah is a genetically-modified autologous T-cell immunotherapy. According to the FDA, each dose of Kymriah is a customized treatment created using a patient’s own T-cells, a type of white blood cell known as a lymphocyte. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

In other news, the FDA also expanded the approval of Actemra (tocilizumab) to treat CAR T-cell-induced severe or life-threatening CRS in patients 2 years of age or older. In clinical trials in patients treated with CAR-T cells, 69 percent of patients had complete resolution of CRS within two weeks following one or two doses of Actemra, FDA says.

Because of the risk of CRS and neurological events, Kymriah is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU). The FDA is requiring that hospitals and their associated clinics that dispense Kymriah be specially certified. As part of that certification, staff involved in the prescribing, dispensing, or administering of Kymriah are required to be trained to recognize and manage CRS and neurological events. 

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. and the expanded approval of Actemra to Genentech Inc.

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