FDA OKs first gene-therapy treatment for young cancer patients – Orange County Register

The U.S. Food and Drug Administration on Wednesday approved the first gene therapy available in the U.S. in a new approach to the treatment of children and young adults suffering from cancer.

The therapy is called Kymriah and it’s made by Novartis AG of Switzerland.

The FDA in a statement said it approved a treatment for cancer patients up to 25 years of age who have relapsed or did not show signs of improvement after treatment for B-cell acute lymphoblastic leukemia.

“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 in the statement. “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. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”

Kymriah is a part of a new drug therapy called genetically-modified autologous T-cell immunotherapy. Each dose is customized using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. The patient’s T-cells are modified to target and kill leukemia cells that have a specific antigen on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells, according to the FDA.

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

The safety and efficacy of Kymriah were demonstrated in one multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was 83 percent.

Novartis has announced a cost for the therapy, according to Reuters, but British health authorities have said a price of $649,000 for a one-time treatment would be justified given the significant benefits, according to a Kaiser Health News report last week.

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