FDA approves new leukemia treatment, costs $475k

Friday, 01 Sep, 2017

Despite these negatives, FDA approval of gene therapy is a big step for the treatment both in the United States of America and around the globe.

The U.S. Food and Drug Administration (FDA) today approved a new cancer therapy that involves genetically modifying a patient's immune cells. It's the first type of gene therapy to hit the US market.

Cell-based gene therapy Kymriah-first of its kind in the United States-will be used to reprogram patients' own cells to attack a deadly disease.

This breakthrough allows scientists to manipulate a patient's blood cells, turning them into an army that can seek and kill cancer cells.

Mitchell said the campaign group thought a price below $300,000 was more appropriate, but said the estimate came with a "huge disclaimer" because Novartis has not shared information about its costs. It's expected to be a 22-day turnaround for Novartis' Kymriah treatment.

"Not only does Kymriah 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", Marks said in the FDA statement. The company is carefully training hospitals and staff to provide the treatment, which can cause a life-threatening immune reaction, as well as long-term complications. Clinical trials of patients treated with CAR-T cells found that 69% had complete resolution of CRS within 2 weeks of one or two doses of tocilizumab.

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

In the study that led to Kymriah's approval, 83% of patients went into remission within three months, according to the FDA. Novartis also considered the cost of bone-marrow transplants, which are now given to many leukemia patients whose cancer relapses. Beyond the cost of the procedure, patients would need to pay for traditional chemotherapy, which is given before vehicle T-cell therapy to improve its odds of success. Kymriah will have a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems. The clinical review was coordinated by the FDA's Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

"Patients who get this therapy basically have no chance to survive", Satwani said.

Cooper said fewer inpatient and outpatient visits for treatment and fewer hospitalizations are likely to result from treatment with auto T-cell therapy, which he said would allow parents and other family members and caregivers "to resume a normal life, including the possibility to return to work". Instead of getting an updated therapy that works better on a disease every decade or so, we might begin to see second-generation cell therapies in just a few years.

Since its founding in 2001, ACGT has allocated approximately $29 million in grants to USA and Canadian cancer gene therapy research projects. "The lessons learned from the clinical trials of vehicle T-cell operations will assist us in being able to provide to the larger population".

With the treatments available now, fewer than 10 percent of patients with the relapsed, hard-to-treat leukemia are alive five years after diagnosis.

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