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Cancer-killing drug approved, but will you be able to afford it?
September 24 2017, 01:22 | Rex Rios
Novartis' CAR-T gene therapy, the first approved by FDA, to be priced based on cancer patients' outcomes
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.
Also on Wednesday, the FDA broadened the approval of Genentech Inc.'s Actemra (tocilizumab) to treat auto (chimeric antigen receptor) T-cell-induced severe or life-threatening cytokine release syndrome in patients 2 years old and up.
Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells.
This procedure, known as Kymriah (tisagenlecleucel), works to combat acute lymphoblastic leukemia (ALL), the most common childhood cancer in the United States with over 3,000 new cases diagnosed every year. Grupp is Emily Whitehead's doctor - she was the first patient to receive the cell therapy, and though she was as close to 48 hours from organ failure when first enrolled in the experimental treatment, Whitehead has now been cancer-free for five years. Kymriah is intended for people with B-cell ALL whose cancer hasn't responded to or has returned after treatment, the FDA said. The modified cells are then transfused back.
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. The worst is cytokine release syndrome, a common immunotherapy complication that causes potentially life-threatening fever and flu-like symptoms.
However, Kymriah comes with the potential for severe side effects.
Scientists often refer to this type of gene therapy as a "living drug" because it involves using genetically modified immune cells from patients. The therapy also can cause neurological events, serious infections, low blood pressure and acute kidney injury. Kymriah's maker, Novartis, hasn't provided a price for the drug.
Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug. The agency said that 69% of patients developing the condition during vehicle T-cell trials "had complete resolution of CRS within 2 weeks following one or two doses" of tocilizumab, which inhibits interleukin-6. Research is therefore focusing for now on refining the therapy for patients of this disease.
Even before Novartis' Kymriah was approved, there was concern about pricing of the new therapy, given that new therapies are typically priced based on level of innovation and cancer is a particularly expensive area.
For more on childhood acute lymphoblastic leukemia, visit the U.S.