Health Desk—July 13, 2017: Novartis AG's pioneering new cancer drug won enthusiastic support from a federal advisory panel on Wednesday, paving the way for approval of the first U.S. gene therapy.
The panel unanimously recommended that the Food and Drug Administration approve the drug, tisagenlecleucel, for patients ages 3 to 25 with relapsed B-cell acute lymphoblastic leukemia (ALL), the most common form of U.S childhood cancer.
The drug uses a new technology known as CAR-T, or chimeric antigen receptor T-cell therapy, which harnesses the body's own immune cells to recognize and attack malignant cells.
In a clinical trial, 83 percent of patients who had relapsed or failed chemotherapy achieved complete or partial remission three months post infusion. After one year 79 percent of patients were still alive.
Patients with ALL who fail chemotherapy typically have only a 16 percent to 30 percent chance of survival.
"This is a potentially paradigm-changing type of benefit," said Dr. Brian Rini, a panelist and physician at Cleveland Clinic Taussig Cancer Institute.
The FDA is not obliged to follow the recommendations of its advisers but typically does so.
However, the agency is expected to rule on the drug by the end of September.