FDA Makes Gene Therapy Available for First Time in USA

Earlier this week, the FDA made gene therapy available for the first time in the United States. This decision, ushers in “a new approach to the treatment of cancer and other serious and life-threatening diseases” said the FDA.

The product, Kymriah (tisagenlecleucel), is for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). According to FDA Commissioner Scott Gottlieb, “[w]e’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer.”

Kymriah is a genetically-modified autologous T-cell immunotherapy. 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 patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses” said Gottlieb. 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 (CBER). “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.”

“There has been an urgent need for novel treatment options that improve outcomes for patients with relapsed or refractory B-cell precursor ALL …. Novartis is collaborating with (Centers for Medicaid Services) to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month. Future potential indications would be reviewed for the most relevant outcomes-based approach,” Novartis, the drug company that makes Kymriah, said in a statement.

On Wednesday, the FDA also expanded approval for another drug, tocilizumab, to treat CAR T-cell induced severe or life-threatening CRS in patients 2 years of age or older. In clinical trials in patients treated with CAR-T cells, 69 percent of patients had complete resolution of CRS within two weeks following one or two doses of tocilizumab.