Novartis AG’s CAR-T cell therapy for leukemia approved by FDA in ‘historic action’; price to be based on outcomes – MarketWatch

Novartis AG's NVS, -0.57% CAR-T cell therapy was approved by the Food and Drug Administration on Wednesday, making it the first gene therapy to be available in the U.S. Novartis' Kymriah was approved for young people up to age 25 with a form of acute lymphoblastic leukemia. CAR-T, or chimeric antigen receptor T-cell therapy, uses a patient's immune T-cells and re-engineers them to better fight cancer. As such, each dose of Kymriah is customized to the individual patient's T-cells through genetic modification. Novartis said on Wednesday that it will work with the Centers for Medicare and Medicaid Services so medicine prices can be "based on the clinical outcomes achieved, which would eliminate inefficiencies from the health care system." For Kymriah, Novartis is also working with CMS "to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month." Novartis did not give any specifics as to what the price range might be. 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. On Wednesday, the FDA also expanded approval of Roche's ROG, +0.29% Genentech's rheumatoid arthritis drug Actemra to treat CAR-T cell-induced cytokine release syndrome, which consists of high fever and flu-like symptoms and can be life-threatening; nearly 70% of patients had CRS completely resolved in two weeks using one or two doses of Actemra, the FDA said. Kite Pharma, which is also working in the CAR-T space, has also been racing to gain FDA approval; the biotech's $11 billion acquisition by Gilead Sciences Inc. GILD, +0.05% was reported earlier this week. Gilead shares surged 5.5% in extremely heavy midday trade. Novartis shares declined 1% in heavy midday trade. Novartis shares have risen 2.8% over the last three months, compared with a 1.6% rise in the S&P 500 SPX, +0.20%

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Novartis AG's CAR-T cell therapy for leukemia approved by FDA in 'historic action'; price to be based on outcomes - MarketWatch