FDA Approves First Gene Therapy in U.S. -- Update

By Austen Hufford Features Dow Jones Newswires

The Food and Drug Administration has approved the first gene therapy in the U.S., a long-heralded move for a promising method to combat cancer.

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The FDA said Wednesday it approved Novartis AG's Kymriah for certain children and young adults who suffer from a form of 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," FDA Commissioner Scott Gottlieb said.

Numerous pharmaceutical companies are investing in gene-therapy projects. This week, Gilead Sciences Inc. agreed to pay about $11 billion for Kite Pharma Inc., in an ambitious bet on the approach.

Kymriah is a type of personalized immunotherapy known as CAR-T, or chimeric antigen receptor T-cell therapy. T-cells, which are a type of white blood cell, are removed from an individual patient and sent to a manufacturing center where they are genetically programmed to target leukemia cells that have a specific marker. The cells are then infused back into the patient to kill cancer cells.

The specific form of cancer, acute lymphoblastic leukemia, impacts bone marrow and blood and is the most common childhood cancer in the U.S. There are about 3,100 patients ages 20 and younger diagnosed with the disease each year, according to the National Cancer Institute. Kymriah is approved for use in the most common form of the disease and is only for patients whose cancer hasn't responded to initial treatments.

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While promising, CAR-T treatments won't be like other drugs that win FDA approval, which can quickly wind up on pharmacy shelves and hospitals. The rollout of this new breed of treatments is currently complicated by unresolved questions.

Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug. In the U.S., only a few dozen specialized hospitals are currently qualified to provide CAR-T treatments, which require retrieving, processing and then returning immune cells to the patient. Novartis said it expects between 30 and 35 centers to be certified to offer the treatment by the end of the year.

Treatment with Kymriah also has the potential for severe side effects, including cytokine release syndrome, which leads to high fever, flu-like symptoms, and other neurological events. The FDA also approved expanded use of Roche Holding AG's Actemra, which treats cytokine release syndrome.

Write to Austen Hufford at austen.hufford@wsj.com

(END) Dow Jones Newswires

August 30, 2017 12:35 ET (16:35 GMT)