FDA Approves CAR T-Cell Therapy for Non-Hodgkin Lymphoma

FDA Approves CAR T-Cell Therapy for Non-Hodgkin Lymphoma

It represents a breakthrough in hematologic cancer treatment in which a patient's own T cells are engineered to seek and destroy cancer cells.

Adults with relapsed or refractory non-Hodgkin lymphoma (NHL) now have new hope. The FDA has approved a risk evaluation and mitigation strategy (REMS) for Yescarta that will inform and educate health care professionals about its associated risks.

Gilead's chief executive John Milligan said the drug's FDA approval was an "important day" for lymphoma patients who had "limited" treatment options. It's the second time the FDA has approved a gene therapy for use in the U.S. following a procedure meant to treat leukemia earlier this year.

The safety and efficacy of the novel gene therapy was investigated in a clinical trial that included more than 100 adults with refractory or relapsed DLBCL.

The therapy includes a boxed warning for cytokine release syndrome and neurologic toxicities, the FDA said.

The drug, named Yescarta, was developed by Israeli-founded Kite Pharma and it is expected to cost some $373,000, likely generating hundreds of millions of dollars in sales.

"In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer", FDA Commissioner Dr. Scott Gottlieb said in a statement.

Novartis, the company that makes Kymriah, is also working to get the drug approved in an aggressive form of lymphoma in adults, which could introduce some competition into the adult market.

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According to Gilead, about 7,500 lymphoma patients each year in the United States would be qualified for CAR-T treatment.

CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer can often evade. The FDA has approved the CD19-directed auto T-cell therapy axicabtagene ciloleucel (axi-cel; Yescarta) for people with large B-cell lymphoma following 2 prior therapies, including for those with diffuse large B-cell lymphoma (DLBCL).

Gilead shares stand at US$80.01 in NY.

Because of these risks, FDA is requiring hospitals and associated clinics that dispense the treatment to be specially certified, including training to recognize and manage CRS and nervous system toxicities.

"We believe this is only the beginning for CAR-T therapies", said Arie Belldegrun, MD, FACS, the founder of the Gilead subsidiary, Kite, that won the approval.

Significantly, it is the first CAR-T drug approved for those patients.

The American Cancer Society estimates that 72,240 people will be diagnosed with non-Hodgkins lymphoma this year and 20,140 people will die from it.

Dr. David Maloney, medical director of cellular immunotherapy at Fred Hutchinson Cancer Research Center, helped develop the therapy and said the FDA's second approval of a CAR-T cell therapy "validates the revolution underway in the field of cellular immunotherapy". While this therapy may be groundbreaking, it comes with potentially lethal side effects.