Novartis prices CAR-T Kymriah at $475K

Childhood Leukemia Will Finally Have An FDA Approved Treatment

Researchers filter the cells from a patient's blood, reprogramme them with a "chimeric antigen receptor" that targets cancer, and grow hundreds of millions of copies.

The immunotherapy now can be used in children and young adults with B-cell ALL that will not respond to other therapies, the FDA announced.

"More research is needed to make this therapy more effective for a broader population, to reduce the severe side effects that patients experience during treatment, and ultimately to find a broader application beyond blood cancers", he said.

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 transforms a patient's immune system cells to fight a form of leukemia.

About 3,100 Americans ages 20 and younger are diagnosed with acute lymphoblastic leukemia each year, and 15 to 20 percent of them don't respond to the initial treatment or have their disease return, according to the National Cancer Institute.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", said FDA Commissioner Scott Gottlieb, MD. It's these patients for whom Kymriah is intended.

In a key study of 63 advanced patients, 83% went into remission, although it is not clear how long the benefit lasts: Some patients did relapse months later.

It's too soon to tell how widely applicable CAR-T will be - it's especially suited to leukemia since infusing patients with T-cells is relatively simple.

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 will carry 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. In addition, clinicians involved in the prescribing, dispensing, and administering of the therapy will need training to recognize these side effects. The price is well below the $700,000 that was estimated before the approval of the drug.

Novartis plans additional filings for Kymriah in the US and EU later this year, including applications with the FDA and European Medicines Agency, for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL).

Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug. More than half were children and teens.

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