Gilead Cell-Therapy Drug Yescarta Gets FDA Approval -- Update

By Jonathan D. Rockoff Features Dow Jones Newswires

Gilead Sciences Inc.'s $11 billion bet on buying Kite Pharma Inc. is poised to pay off, with the approval Wednesday of Kite's flagship cell-therapy treatment for adults with advanced lymphoma.

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The therapy, known as a Car-T treatment, uses genetically engineered T-cells to attack the blood cancer. It is the second such therapy to get a regulatory green light, after Novartis AG's Car-T drug Kymriah was approved in late August for a form of leukemia in children and young adults.

Like Novartis's therapy, which was priced at $475,000, Gilead's treatment commands a six-figure list price: $373,000.

Gilead Chief Executive John Milligan said in a statement that the approval marks "an important day for patients with relapsed or refractory large B-cell lymphoma who have run out of options and have been waiting for new treatments that may help them in their fight against cancer."

The newly approved therapy, dubbed Yescarta, was the crown jewel of Gilead's recent purchase of biotech Kite Pharma. It has been among the most highly anticipated new drugs on Wall Street, which estimates the therapy will have $1.7 billion in world-wide sales in five years, according to EvaluatePharma, a market-research firm.

Sales of Yescarta should bolster Gilead's efforts to diversify beyond its legacy drugs for HIV/AIDS and hepatitis C as their sales slow.

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The company, which was criticized by patients, doctors and lawmakers for the high list price of hepatitis C treatment Sovaldi, could face a new round of scrutiny over Yescarta's price tag. A Gilead spokeswoman pointed to clinical-trial data showing the drug worked in far more patients than current standard-of-care treatment.

"Taking into account these important clinical data, we conducted extensive research with commercial and government payers, as well as targeted cancer centers, to set a price that reflects the value represented by this innovation, and that supports accessibility of this personalized therapy," the Gilead spokeswoman said.

The Food and Drug Administration approved Yescarta for adults with certain advanced forms of diffuse large B-cell lymphoma, the most common type of non-Hodgkin lymphoma in adults. Gilead said about 7,500 patients in the U.S. will be eligible each year for treatment.

Cell therapies such as Yescarta are part of a broader wave of immunotherapies that harness the body's immune system to attack cancer, providing a new option for certain cancer patients whose disease has proven hard to treat.

The new treatments have "gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer," FDA Commissioner Scott Gottlieb said in a statement.

Yescarta is made from a patient's own immune cells, called T cells, which are taken from the patient and sent to a laboratory where they are genetically engineered to attack cancer. The modified T cells are put back into the patient where they are able to recognize and attach to a particular protein on tumor cells, killing them. The new feature that gives the engineered T cells their cancer-fighting powers is called a chimeric antigen receptor, hence the name Car-T therapy.

In the pivotal clinical trial of Yescarta, tumors shrank in 72% of the 101 subjects and disappeared in 51% of the patients, according to Gilead.

Side effects reported in the trial included fever, anemia and low white-blood cell counts, which can increase vulnerability to infection. The deaths of two subjects were determined to be related to the drug's use.

Yescarta will have a Black Box warning regarding the risk of "life-threatening" high fever and flulike symptoms during treatment, and use will be limited to specially certified hospitals and cancer clinics following risk-mitigation guidelines for safe use, according to the FDA announcement.

Write to Jonathan D. Rockoff at Jonathan.Rockoff@wsj.com

(END) Dow Jones Newswires

October 18, 2017 19:04 ET (23:04 GMT)