The Food and Drug Administration approved the therapy from Novartis called Kymriah, in children and young adults with acute lymphoblastic leukemia whose disease has come back in spite of previous treatments.
This article first appeared August 30, 2017 on Kaiser Health News.
By Liz Szabo
The country’s first approved gene therapy — approved Wednesday to fight leukemia that resists standard therapies — will cost $475,000 for a one-time treatment, its manufacturer announced.
Switzerland-based Novartis, which makes the innovative therapy, announced that the drug will cost nothing if patients fail to benefit in the first month.
The Food and Drug Administration approved the therapy, called Kymriah, in children and young adults with acute lymphoblastic leukemia whose disease has come back in spite of previous treatments. These patients typically have a poor prognosis, surviving three to nine months, according to Novartis.
In the study that led to Kymriah’s approval, 83 percent of patients went into remission within three months, according to the FDA. Novartis estimates about 600 patients a year would be eligible for the treatment, which belongs to a class of drugs known as CAR T-cell therapies.
Kymriah treats cancer in an entirely new way. The individualized approach involves harvesting cancer patients’ immune cells, genetically engineering them, then returning them to patients’ bodies. The genetic engineering process aims to rev up patients’ immune systems to better fight cancer.
Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.