Medicine

FDA Approves First Gene Therapy in the United States

FDA Approves First Gene Therapy in the United States

The U.S. Food and Drug Administration (FDA) today approved a new cancer therapy that involves genetically modifying a patient's immune cells.

The treatment, to be sold under the name Kymriah, was approved for patients up to 25 years of age who have relapsed or were not helped by initial treatment for B-cell acute lymphoblastic leukemia.

Also on Wednesday, the FDA broadened the approval of Genentech Inc.'s Actemra (tocilizumab) to treat vehicle (chimeric antigen receptor) T-cell-induced severe or life-threatening cytokine release syndrome in patients 2 years old and up.

"We're entering a new frontier in medical innovation with the ability to reprogramme a patient's own cells to attack a deadly cancer", said FDA commissioner Scott Gottlieb. 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.

Based on available data, patients on the treatment have had an 89% chance of surviving at least six months and a 79% chance of surviving at least a year, with most being relapse-free at that point.

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In this type of therapy, patients' own immune cells are modified ex vivo to include a gene for the auto protein, which directs them to attack leukemia cells carrying the CD19 antigen.

ALL is the most common type of cancer among children, according to the National Cancer Institute. The modified cells are then transfused back. "At the FDA, we're committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving", he said. "Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease", Peter Marks, MD, PhD, direct of the Center for Biologics Evaluation and Research, FDA, said in the release. In a trial of Kymriah, 83 percent of 63 patients with hard-to-treat disease were in remission within three months, according to Novartis. Novartis said it expects between 30 and 35 centers to be certified to offer the treatment by the end of the year.

However, tisagenlecleucel (also known as CTL019) can cause severe side effects, particularly a condition called cytokine release syndrome (CRS) that can become life-threatening.

Novartis is required to conduct followup study to assess the safety of the treatment long-term.


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