ODAC approval of Novartis' CAR T-Cell therapy paves the way for its FDA approval as a commercially available treatment for B-cell ALL.
It looks like 2017 will be a transformative year for CAR-T therapy. The US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) unanimously recommended approval of CTL019 (tisagenlecleucel) on July 12, 2017 for the treatment of relapsed or refractory pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL).
Tisagenlecleucel is an investigational chimeric antigen receptor (CAR) T cell therapy by Novartis. The advisory committee hearing was the last major regulatory milestone before the agency decides in September whether to approve the treatment, which would make this the first-ever commercially approved CAR-T cell therapy. The committee’s unanimous positive vote bodes well for this gene therapy approach.
Effective treatment options for patients with relapsed/refractory ALL are limited. In pediatric and young adult patients with B-cell ALL who relapse or are refractory to treatment, the survival rates are very low. "We know firsthand from treating children and young adults with relapsed/refractory B-cell ALL that they desperately need innovative medicines that provide a new approach to managing this aggressive disease," said Stephan Grupp, MD, PhD, Professor of Pediatrics at the Perelman School of Medicine at Pennsylvania University and Director of the Cancer Immunotherapy Frontier Program, both in Philadelphia, Pennsylvania.
CTL019 was first developed by the University of Pennsylvania and uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular responses. This approach in clinical studies has been associated with long-lasting remissions in relapsed and treatment refractory patients.
CAR-T is manufactured for each individual patient using their own cells. During the treatment process, T cells are drawn from a patient's blood and reprogrammed in the manufacturing facility to create T cells that are genetically coded to express a chimeric antigen receptor to recognize and fight cancer cells and other B-cell malignancies.