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United States approves first gene therapy treatment

31 August 2017

Novartis (NVS) was the first drugmaker to cross the finish line in developing cancer treatments known as CAR-T drugs Wednesday after the Food and Drug Administration approved its drug, Kymriah, for children and adults with a type of bone marrow cancer.

This first use of CAR-T therapy is aimed at patients desperately ill with acute lymphoblastic leukaemia, which strikes more than 3,000 children and young adults in the U.S. each year.

The approval of Novartis' CAR-T therapy comes just two days after Gilead Sciences Inc. announced an agreement to spend $11.9 billion and buy Kite Pharma Inc., which is developing its own CAR-T therapy scheduled for an FDA ruling by November 29.

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"We're entering a new frontier in medical innovation with the ability to reprogramme a patient's own cells to attack a deadly cancer", said FDA commissioner Scott Gottlieb.

Novartis has not yet announced the cost for the therapy, 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.

What's perhaps most exciting about CAR-T is that it operates similarly to a vaccination, providing the patient with lifetime protection against the targeted cancer.

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Kymriah, which is also known as CTL019, is a type of CART-T therapy that involves immune cells being extracted and genetically modified to destroy cancer before it returns to the body.

About 3,100 patients 20 and younger are diagnosed with ALL each year, according to the U.S. National Cancer Institute. More than half were children and teens.

"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 statement. They are then reintroduced back into the patient to do battle against the tumor. Kymriah will carry a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems.

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The company said it plans to work with the Centers for Medicare and Medicaid Services to implement the pricing strategy. "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".

United States approves first gene therapy treatment