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First gene therapy coming to U.S. after FDA approval

CAR T-cell therapy approved to treat certain children and young adults with B-cell acute lymphoblastic leukemia.

By
Amy Wallace
The U.S. Food and Drug Administration today approved the first gene therapy treatment in the United States to treat relapsing acute lymphoblastic leukemia. Photo by Anawat Sudchanham/Shutterstock
The U.S. Food and Drug Administration today approved the first gene therapy treatment in the United States to treat relapsing acute lymphoblastic leukemia. Photo by Anawat Sudchanham/Shutterstock

Aug. 30 (UPI) -- The U.S. Food and Drug Administration announced today it is making the first gene therapy available in the United States, an immunotherapy treatment for acute lymphoblastic leukemia.

The FDA approved Kymriah, or tisagenlecleucel, for certain pediatric and adult patients diagnosed with a form of acute lymphoblastic leukemia, or ALL.

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Kymriah is a cell-based gene therapy approved for the use of treatment of patients up to age 25 with B-cell precursor ALL that is in a second or later relapse.

"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease," Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, said in a press release. "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."

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Kymriah is a genetically-modified autologous T-cell immunotherapy with each dose customized using the patient's own T-cells. The altered T-cells include a new gene that contains a protein called chimeric antigen receptor, or CAR, that directs the T-cells to kill leukemia cells with the antigen CD19.

The treatment was tested in a clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor 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," FDA Commissioner Dr. Scott Gottlieb, said. "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."

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