Groundbreaking Leukemia Gene Therapy Carries $475k Price Tag

Ray Weaver
September 1, 2017

- The US Food and Drug Administration approved a new leukemia treatment, which the agency considers the first gene therapy it has cleared to hit the market in the United States.

"We're excited and proud to have moved this vehicle therapy, in collaboration with Novartis and CHOP, through all phases of development and clinical trials, established its efficacy, and now extended its reach to children across the country under this FDA approval", he added.

The FDA today also expanded the approval of Actemra (tocilizumab) to treat auto T-cell-induced severe or life-threatening CRS in patients 2 years of age or older. The treatment was originally developed by researchers at the University of Pennsylvania and licensed to Novartis. The FDA approval of this therapy is based on the results of the Phase II ELIANA trial, which was sponsored by Novartis and included 25 centres in the US, EU, Canada, Australia and Japan.

"While Novartis's decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive", president of Patients for Affordable Drugs, David Mitchell, told The Guardian. Stat News reported on Wednesday that Novartis' head of oncology, Bruno Strigini, said in a conference call that the $475,000 price for Kymriah was an attempt to "balance patient access to the drug with ensuring a return on the company's investment".

Novartis will create a registry to follow patients for 15 years after being treated to monitor their progress and any potential, future side effects. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

The trial was successful, with 83% of the 63 clinical patients treated for relapsed or refractory B-cell undergoing successful remission within three months of treatment. 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 established path to commercialization taken by Novartis will provide considerable motivation for cell therapy scientists and development companies as well as investors looking to benefit from the ongoing success in the industry". The therapy involves harvesting a patient's T-cells and then genetically modifying them to hold potent molecules called chimeric antigen receptors (CARs). About half of the patients in a Kymriah study got cytokine-release syndrome, a response to the reprogrammed cells running loose in the body. Satwani - who hasn't delivered the CAR-T therapy but is heading up the program at Columbia, one of the 32 sites Novartis will use - said patients would need to be monitored closely for about a month, meaning they'd need to be less than a two-hour drive from their hospital. About 3100 people under age 21 are diagnosed with ALL each year in the United States, though most respond to standard therapy. 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. CAR-T's side effects can be deadly.

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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