Cancer Gene Therapy Gets Green Light - And $475000 Price Tag

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

01 September, 2017

Since the therapy is made from a person's own immune system, the process can take about three weeks. After re-engineering them to attack cancer, frozen cells are returned a couple weeks later for reinfusion into patients.

The rights to the new cancer treatment were given to them and they would sell it under the label Kymriah for $475,000. But they have not altered the actual DNA of the patients' cells like Kymriah is touted to do.

While that is the only time the treatment's given, it's not the end of the road.

For now, Kymriah is only available to a small subsection of cancer patients.

Many patients in the ALL trials experienced a side effect called cytokine release syndrome (CRS) including grade 3 or grade 4, which includes varying degrees of flu-like symptoms, with high fevers, nausea, and muscle pain, and temporary neurologic symptoms, including delirium, but also low blood pressure and breathing difficulties requiring ICU-level care in the most severe cases.

The price may be high for vehicle T-cell therapy, but it is important to weigh the costs against the potential benefits for patients, Gwen Nichols, MD, chief medical officer for the Leukemia & Lymphoma Society, said in an interview with OncLive. Its approval comes after an FDA advisory committee backed the drug by a unanimous vote last month.

The therapy does have side effect for which the FDA has created a treatment. The treatment is called a living drug because involves using genetically modified immune cells from patients to attack their own cancer. But while the treatment is complex, it offers huge rewards.

"Recognising our responsibility we set the price below that level", said Strigini. "It's a great accomplishment".

Novartis, the pharmaceutical company behind the treatment, saw complete remission in 83% of 63 patients who had B cell acute lymphoblastic leukemia. "Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more", said David Mitchell, Founder and President of Patients For Affordable Drugs.

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Cancer cells are seen on a large screen connected to a microscope at the CeBit computer fair in Hanover, Germany, March, 6, 2012.

Another important issue is how Novartis' decision might affect Gilead's approach to pricing its CAR-T drug, Axi-Cel, acquired in the company's Monday purchase of Kite Pharma. The genetic changes wrought through the customized process creates coding for a protein known as a chimeric antigen receptor, or vehicle.

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

Those updates could potentially expand the number of patients cell therapies are able to treat.

Anderson noted that, if Novartis treated 100% of the 600-per-year patient population successfully, that would amount to about $300 million in annual sales-far from blockbuster status, and for a drug that's very expensive to make.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells.

His current research is focused on similar reprogramming of cells but for the detection of lung cancer cells.

"CAR T-cells are not worse than these".

In clinical trials, CAR-T therapies have shown remarkable efficacy against blood cancers.

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