FDA Approves Another Amazing And Costly Cancer-Killing Gene Therapy

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FDA Approves Another Amazing And Costly Cancer-Killing Gene Therapy

Gene therapies are promising for cancer.

In 2010, Josh Feldman was on his honeymoon — a globe-spanning trip that at one point involved swimming with humpback whales — when he noticed a “Garbanzo-sized” lump on the back of his neck. It turned out to be lymphoma, which worsened even as he cycled through treatments: chemotherapy, the immunotherapy Opdivo, a targeted drug called Zydelig. What actually worked for the 55-year-old photographer was a radical treatment in which his own white blood cells were genetically re-engineered to kill his cancer. Compared to the other drugs, it was “a walk in the park,” he said earlier this year while overlooking a mango tree on his property. “I’d like to see it become first-line therapy,” he said. In September, 15 months after his treatment, he was still in remission.

That’s still a long way away. But the treatment Feldman received was just approved by the Food and Drug Administration under the brand name Yescarta, with a bracing price tag: $373,000 per patient. That doesn’t include the costs of dealing Yescarta’s side effects, which include life-threatening fevers triggered by the immune response the therapy creates that can mean weeks in the hospital, or other drugs patients must receive.

FDA Commissioner Scott Gottlieb, himself a cancer survivor, called Yescarta “another milestone in the development of a whole new scientific paradigm.” David Maloney, a physician at the Fred Hutchinson Cancer Research Center, had this to say about Yescarta’s approval in a prepared statement: “There will likely be thousands of lives saved in the next few years because of it.” Louis DeGennaro, the chief executive of the Leukemia and Lymphoma Society, which helped fund Yescarta’s development, wrote in an email blast that “many patients who were out of treatment options are now in remission” because of experimental studies of the drug.

It’s no surprise that the excitement comes at a high economic price. In August, the treatment’s developer, Kite Pharmaceuticals, was purchased by biotech giant Gilead Pharmaceuticals for $11.9 billion, a price which made Kite founder and chief executive Arie Belldegrun’s shares worth some $600 million. The process of making the drug is laborious and expensive. But critics are likely to counter that much of the work of developing the drug was done by the National Cancer Institute, by Steven Rosenberg, a researcher there.

Yescarta is the second treatment of its kind, called a Chimeric Antigen Receptor T-Cell, or CAR-T, for the particular genetic modifications made to white cells. The first, Kymriah, for Novartis, was addressed at children with acute lymphoblastic leukemia, a small group of patients. Yescarta is being approved for a much larger population. About 24,000 Americans are diagnosed with diffuse large-cell b-cell lymphoma (DLBCL), the variety Yescarta treats, every year.

Novartis priced Kymriah at $475,000, which makes Kite’s price sound significantly lower. But Novartis said it is offering insurance companies a unique, “value-based” pricing structure, in which it only pays for those patients who go into remission withing three months. For Novartis’ treatment in DLBCL, that’s only 45% of patients, which means at the $475,000 price, Novartis would only be receiving about $200,000 per patient. (Novartis has not said how it will price in DLBCL).

In patients’ whose disease did not shrink after chemotherapy, Yescarta shrank tumors 70% of the time. More impressively, 51% went into complete remission, meaning their cancer was undetectable. The severe fever and neurological side effects led the FDA to give the treatment a boxed warning, its most severe.

Kite also boasts about its ability to manufacture individual CAR-T cells for each patient. “Our manufacturing success rate was 99% and the turnaround had a median of 17 days,” said David Chang, Kite’s chief scientific officer. “I haven’t anything close to that coming from Novartis.”

In an interview conducted before the approval, Chang also had a response for critics who point to the role that the NCI and the Leukemia and Lymphoma Society had in developing Yescarta as an argument that it should cost less. He says that people don’t have enough appreciation of the difficult of moving from government-sponsored research to an approved, manufactured product.

Read full article: FDA Approves Another Amazing And Costly Cancer-Killing Gene Therapy

Read Full Article: FDA Approves Another Amazing And Costly Cancer-Killing Gene Therapy

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