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Cancer gene-altering therapy gets approved in the United States

A new cancer therapy that involves genetically modifying a patient’s immune cells was just approved by the United States Food and Drug Administration (FDA), marking the first gene therapy treatment approved in the United States.
WebMD Gene Therapy

Gene therapy

The new treatment, produced by Novartis and branded Kymriah, involves removing immune cells called T cells from a patient’s blood and giving them a gene for a protein called a chimeric antigen receptor, or CAR, that directs the T cells to target leukemia cells. The modified cells are then transfused back.

Although the treatment can sometime causes severe side effects, in a Novartis clinical trial the drug caused remission in 83% of 63 young patients with a certain type of leukemia, B cell acute lymphoblastic leukemia (“ALL”).

FDA approval

The FDA approved Kymriah (tisagenlecleucel) for children and adults up to age 25 with B cell who have not responded to conventional therapy or who have relapsed. About 3100 people under age 21 are diagnosed with ALL each year in the United States, though most respond to standard therapy.

Coming soon to the China market?

China has already approved another gene therapy cancer drug, hence the country’s State Food and Drug Administration (SFDA) would likely approve Kymriah now that the Novartis drug has been approved by the United States FDA.

Whether and how strongly Novartis will market the Kymriah in China remains a key question. One primary consideration is pricing — Kymriah will hit the United States market at a cost of USD $475,000. Although this price is reportedly less than expected, it probably places the drug out of reach for most cancer patients in China. Moreover, typical pharmaceutical economies of scale are unlikely to apply to Kymriah, as the modified cells must be made specifically for each patient in special production facilities. In fact, Kymriah supply shortages already arose at some medical centers during clinical trials.

4 Responses to “Cancer gene-altering therapy gets approved in the United States”

  1. 三五游戏
    September 7, 2017 at 4:20 pm #

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  2. 沃八达
    September 25, 2017 at 3:48 pm #

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  3. 套图网
    October 18, 2017 at 3:20 pm #

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  4. 套图网
    October 25, 2017 at 7:59 am #

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