The U.S. Food and Drug Administration on Wednesday approved Novartis AG's (NVS - Get Report) Kymriah, a cell-based gene therapy for certain children and young adults with B-cell acute lymphoblastic leukemia.
This marks the first gene therapy greenlighted by the FDA. The action comes after an FDA advisory panel last month recommended that the agency approve the treatment.
"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, M.D. in Wednesday's announcement.
"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease," said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research, in the news release. "Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials."
In its announcement, Novartis said it plans to make additional filings for Kymriah in the U.S. and EU 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. The company also said it anticipates making additional filings beyond the U.S. and EU next year.
Novartis' American depository shares were trading at $82.83 on Wednesday, down 0.9%.
Also on Wednesday, the FDA broadened the approval of Genentech Inc.'s Actemra (tocilizumab) to treat CAR (chimeric antigen receptor)T-cell-induced severe or life-threatening cytokine release syndrome in patients 2 years old and up.
Genentech is a unit of Roche Holding Ltd.
—Bill Meagher contributed to this article
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