Historic FDA Approval Makes First Gene Therapy Available in US

Американцы проведут рискованную генную модификацию человека

CAR-T therapy can cause serious side effects

Connie Andrews | 31 August, 2017, 06:34

Each dose of Kymriah is created using the person's own immune system T-cells.

The treatment will be sold under the name Kymriah, and is approved for children and young adults up to age 25 with cancers that don't respond to traditional treatment, or who are suffering from a second relapse, a broad indication that will give more patients access to the novel technology.

Novartis, which won a priority review voucher alongside the approval, noted Kymriah will be manufactured for each individual patient using their own cells at a facility in Morris Plains, New Jersey, where the company says it has designed a manufacturing and supply chain platform that allows for an individualized treatment approach on a global scale.

"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", Gottlieb said.

In the main study that informed the advisory committee's decision in July, roughly half of 68 patients experienced high-grade CRS, though none died from it. The treatment reprograms a patient's T cells to carry a "chimeric antigen receptor" that targets cancer cells. It progresses quickly, and is the most common childhood cancer in the United States, affecting an estimated 3,100 patients aged 20 years and younger annually. The company will also offer financial assistance for some cases, but they didn't promise all patients will be able to get the therapy.

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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. But if it's the last treatment patients need, "that's a really significant one-time investment in their wellness, especially in kids who have a whole lifetime ahead of them".

One study cited in the FDA's announcement treated 63 children and young adult patients with Kymriah, which saw an 83 percent remission rate after three months.

The FDA acknowledged Kymriah's potential for severe side effects by approving the treatment with a boxed warning for cytokine release syndrome (CRS), a systemic response to the activation and proliferation of vehicle T-cells causing high fever and flu-like symptoms, and for neurological events.

Therapies that would operate in similar ways - engineering the immune system's T cells to fight disease more effectively - are under investigation for a host of other conditions, including HIV/AIDS, genetic and autoimmune disorders and other forms of cancer. Auto directs the T cells to target and attack leukemia cells that have CD19 on the surface. A drug called tocilizumab can often successfully treat those complications.

Novartis representatives said they calculated a "cost-effective price" for the therapy that fell between $600,000 and $750,000. "Today's approval of Actemra/RoActemra for CRS provides physicians with an important tool to help manage this potentially life-threatening side effect".

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Historic FDA Approval Makes First Gene Therapy Available in US

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