CAR-T cell therapy CTL019 gains FDA Breakthrough Therapy

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to CTL019 (tisagenlecleucel-T), an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of adult patients with relapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who have failed two or more prior therapies.

This is the second indication for which CTL019 has received this designation; the first being for the treatment of r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients, says the product’s developer, Swiss pharma giant Novartis (NOVN: VX).

Last month, the FDA accepted Novartis’ Biologics License Application (BLA) filing and granted priority review for CTL019, 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). It is in a race with rivals including Kite Pharma to become the first to secure regulatory approval for a CAR-T treatment.