FDA Approves First Gene Therapy For Leukemia

Novartis was the first to receive FDA approval for a CAR-T drug to treat cancer

Novartis was the first to receive FDA approval for a CAR-T drug to treat cancer

Novartis AG on Wednesday won highly anticipated USA approval for the first of a new type of potent gene-modifying immunotherapy for leukemia, a $475,000 treatment that marks the start of a potential new treatment paradigm for some cancers. Tisagenlecleucel (Kymriah) is a personalized genetically-modified autologous T cell immunotherapy created to use a patient's own T cells to target and kill leukemia cells with CD19 antigens on the surface, according to a press release.

The approval allows for Kymriah - the name that the cell-based gene therapy will be sold under - to be used by children and other young patients up to the age of 25 who suffer from B-cell precursor acute lymphoblastic leukemia (ALL).

The approval was widely expected after an FDA advisory panel last month unanimously recommended the action.

In addition to Kymriah's approval, FDA also announced the expanded approval of Roche's Actemra (tocilizumab) to treat auto T-cell-induced severe or life-threatening cytokine release syndrome in patients 2 years of age or older.

The National Cancer Institute estimates 3,100 patients are diagnosed each year with ALL, which can come in several forms.

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Novartis Pharmaceuticals announced the price for its one-time infusion would be $475,000, but noted there would be no charge for patients who didn't show a response within a month of receiving the treatment. The company will also offer financial assistance for some cases, but they didn't promise all patients will be able to get the therapy. Tisagenlecleucel carries a boxed warning for cytokine release syndrome (CRS) and neurological events, which can be life threatening. "We hope the momentum behind the technology builds as we continue to investigate the abilities of personalized cellular therapeutics in blood cancers and solid tumors to help patients with many other types of cancer", said Dr. Stephan Grupp, director of the cancer immunotherapy frontier program at University of Pennsylvania and who led the studies on children with ALL. Some 83 percent of those given the therapy were in remission within three months of treatment, the agency said.

All patients who had CR or CRi were associated with negative minimal residual disease status in the bone marrow. Each dose of Kymriah is a customized treatment created using an individual patient's own T-cells, a type of white blood cell known as a lymphocyte.

The Food and Drug Administration called the approval historic.

No fewer than 76 CAR-T treatments are now under review at the FDA, and Gottlieb predicted that other approvals would follow. Also called Kymriah, the treatment isn't actually a drug in the traditional sense, but a collection of the patient's own cells that have been removed from the body and genetically engineered to recognize and destroy cancer cells circulating in the blood. It's shown very encouraging results in clinical trials, but the price tag will be hefty: Analysts say it will cost "a fortune," or maybe $700,000 for one course of treatment.