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Clients in the News – Novartis Receives Historic Approval for First Gene Therapy in the U.S.

The U.S. Food and Drug Administration (FDA) has approved tisagenlecleucel (Kymriah) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

The historic action makes Novartis Pharmaceutical’s CAR-T cell treatment the first gene therapy available in the United States. (Gilead just negotiated to buy Kite Pharma, which also has a CAR-T therapy under review.) The announcement ushers in a new approach to the treatment of cancer and other serious and life-threatening diseases by using the patient’s own modified T-cells to fight disease.

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

The therapy is approved for patients up to 25 years for ages with B-cell precursor ALL that is refractory or in second or later relapse.

Each dose is a personalized treatment using the patient’s T-cells. After being collected, the T-cells are genetically modified to include a new gene that contains a specific protein that directs the T-cells to target and kill the leukemia cells that have a specific antigen on the surface. Once modified, the cells are infused back into the patient to kill the cancer cells.

The treatment has an overall remission rate with of 83 percent within three months of treatment.

“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.”

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