Novartis announces first CAR-T cell therapy BLA for pediatric and young adult patients with r/r B-cell ALL granted FDA Priority Review

Novartis International AG / Novartis announces first CAR-T cell therapy BLA for pediatric and young adult patients with r/r B-cell ALL granted FDA Priority Review . Processed and transmitted by Nasdaq Corporate Solutions. The issuer is solely responsible for the content of this announcement.

• Priority review for investigational CTL019 (tisagenlecleucel-T), a novel therapy that is manufactured for each individual patient using their own T cells
• Novartis made an early commitment to the emerging field of CAR-T investigational therapies through collaboration with the University of Pennsylvania including CTL019
• Novartis plans to submit an application for market authorization with the European Medicines Agency (EMA) later this year

Basel, March 29, 2017 - Novartis announced today that the US Food and Drug Administration (FDA) has accepted the company's Biologics License Application (BLA) filing and granted priority review for CTL019 (tisagenlecleucel-T), an investigational chimeric antigen receptor T cell (CAR-T) therapy, in relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). This is the first BLA submission by Novartis for a CAR-T. The priority review designation is expected to shorten the anticipated review time by the FDA.

CAR-T is different from typical small molecule or biologic therapies currently on the market because it is manufactured for each individual patient. During the treatment process, T cells are drawn from a patient's blood and reprogrammed in the laboratory to create T cells that are genetically coded to hunt the patient's cancer cells and other B-cells expressing a particular antigen.

"With CTL019, Novartis is at the forefront of the science and development of immunocellular therapy as a potential new innovative approach to treating certain cancers where there are limited options," said Vas Narasimhan, Global Head of Drug Development and Chief Medical Officer, Novartis. "The priority review and file acceptance of CTL019 by the FDA brings us one step closer to delivering this novel treatment option to children and young adults with r/r B-cell ALL in the United States."