Cutting-Edge Pediatric Cancer Therapy Approved by FDA

Novartis’ Kymriah (tisagenlecleucel) has been approved by the U.S. Food and Drug Administration (FDA) to treat pediatric acute lymphoblastic leukemia. The personalized treatment, called CAR (chimeric antigen receptor) T-cell therapy, is a cancer immunotherapy that harnesses the body’s immune system by removing a person’s cells, reengineering them with the drug and then replacing them into the body to attack cancer cells. According to Novartis, the one-time treatment costs $475,000; however, the company is working with the Centers for Medicare and Medicaid Services (CMS) to develop a payment system that reflects how well the drug works in a person. Under that system, CMS pays “only when pediatric and young adult patients respond to Kymriah by the end of the first month.”

“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 Scott Gottlieb, FDA commissioner. “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.”