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FDA Approves First CAR-T Cell Therapy

01 September 2017, 01:49 | Nellie George

Novartis' CAR-T gene therapy, the first approved by FDA, to be priced based on cancer patients' outcomes

Why bluebird bio Inc Jumped Higher Today

The Food and Drug Administration (FDA) has called this a historic moment because it is considered to be the first gene therapy hitting the USA markets. The Food and Drug Administration (FDA) has qualified the treatment as historic since it is the first gene therapy in the U.S market. This revised forecast encompasses the expected cross-industry reaction to the approval, and envisions the next stages in the development of cell therapies and the industry.

Shares of Juno Therapeutics Inc, which past year reported a handful of patient deaths during trials of its CAR-T therapy, were down 8.9 percent at $39.92. Novartis estimates about 600 patients a year would be eligible for the treatment, which belongs to a class of drugs known as vehicle T-cell therapies. Mitchell said to The Guardian that he and his team met with Novartis the day before the drug's approval and price were to be announced, and he claims that the drug company "spent most of the meeting explaining why it needs to charge an astronomical price".

The pharma exec also told Herper he hopes the discussion about Kymriah's cost could lead to a change in the way patients are charged for life-saving treatments. "Not only does [tisagenlecleucel] provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials".

Other severe side effects can pop up and require hospitalization.

Now, following the FDA's announcement, those patients, their families and their oncologists may decide to pursue gene therapy, which starts with the patient's own T-cells and uses genetic engineering to make them more active than usual and better at targeting tumors. Most ALL patients respond to initial therapy, but Kymriah will be an invaluable back-up option for relapsed and refractory patients who now have poor prognosis.

The drug, called Kymriah, is a highly personalized cancer treatment called vehicle T-cell therapy (CAR is short for chimeric antigen receptor).

FDA commissioner Scott Gottlieb said the approval ushers in "a new frontier in medical innovation", adding: "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". Several recent patient deaths in CAR-T clinical trials have raised serious questions over the safety of the treatment.

"Patients who get this therapy basically have no chance to survive", he said. For others, Kymriah serves as a "bridge to transplant", keeping them alive longer enough to undergo this therapy.

Epstein said he envisions cell therapies having much shorter life cycles than traditional drugs.

The U.S health officials have approved a treatment which genetically turns the patient's blood cells into assassins seeking to destroy leukemia.

Doctors don't yet know what the full range of long-term side effects will be.

Over the last few years, CAR-T cell research has exploded, and there are now nearly 300 clinical trials underway experimenting with the treatment. Penn and Novartis are also investigating the next generation of vehicle therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer.

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