Historic FDA Approval Makes First Gene Therapy Available in US

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Novartis has not yet announced the cost for the therapy, but British health authorities have said a price of $649,000 for a one-time treatment would be justified given the significant benefits, according to a Kaiser Health News report last week. Safety and efficacy of tisagenlecleucel were demonstrated in a multicenter clinical trial comprising 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL. A chimeric antigen receptor (CAR) receptor is added to them, and the enhanced T-cells are then returned to the patient's blood stream, where they're newly capable of hunting down and destroying cancer cells.

The treatment will be sold under the name Kymriah, and is approved for children and young adults up to age 25 with cancers that don't respond to traditional treatment, or who are suffering from a second relapse, a broad indication that will give more patients access to the novel technology.

About 3,100 patients 20 and younger are diagnosed with ALL each year, according to the U.S. National Cancer Institute.

What's perhaps most exciting about CAR-T is that it operates similarly to a vaccination, providing the patient with lifetime protection against the targeted cancer.

Each dose of Kymriah contains a patient's own immune cells, which are sent to a lab to be genetically modified using a virus. It's these patients for whom Kymriah is intended.

While promising, CAR-T treatments won't be like other drugs that win FDA approval, which can quickly wind up on pharmacy shelves and hospitals.

Kymriah comes with a boxed warning for neurological events and cytokine release syndrome (CRS), which can both be life-threatening. In addition, clinicians involved in the prescribing, dispensing, and administering of the therapy will need training to recognize these side effects. After three months of treatment, 83 percent of the patients remained cancer-free.

The drug Kymriah was developed by Novartis Pharmaceuticals and the University of Pennsylvania.

Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug.

Other genetic therapies for cancer are also in the research pipeline. Gilead shares were up more than 5 percent on Wednesday as Kite is widely seen as likely to receive the next USA approval of a CAR-T therapy.

"The theory is they should attack the tumor and continue to grow to become a long-term monitoring and treatment system", Lichtenfeld said.

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