A New Approach to Treat Childhood Leukemia: Novartis’ CAR-T Therapy image

The Journal of Law, Medicine & Ethics, January 10, 2018
Frazer A. Tessema and Jonathan J. Darrow (Student Fellow Alumnus)


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From the article:

On August 30, 2017, the US Food and Drug Administration (FDA) announced the approval of tisagenlecleucel (Kymriah; CTL019), Novartis' new treatment for B-cell acute lymphoblastic leukemia (ALL) in children or young adults who are either unresponsive to traditional treatments or whose cancers return after periods of remission.1 Prior to approval, the drug received priority review and breakthrough designations, and had the strong support of the FDA's Oncologic Drugs Advisory Committee (ODAC), which on July 12, 2017 unanimously voted to recommend that the drug be approved.2

Even before it was approved, media outlets, ODAC, and FDA officials offered high praise for the drug, calling it an “unprecedented” and “paradigm changing” treatment that was “ushering in a new era in treating children with relapsed and refractory ALL.”3 In a press release, the FDA described tisagenlecleucel's approval as a historic action that makes available the first gene therapy in the United States.4


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