Novartis Personalized Cell Therapy Receives FDA Breakthrough Therapy Designation

Novartis reported that FDA granted breakthrough therapy status to CTL019, an investigational chimeric antigen receptor (CAR) therapy for the treatment of pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia (r/r ALL). The breakthrough therapy filing was submitted by the University of Pennsylvania's Perelman School of Medicine (Penn), which has an exclusive global agreement with Novartis to research, develop, and commercialize personalized CAR T-cell therapies for the treatment of cancers. Researchers at Penn noted strong, positive early data in pediatric and adult patients with r/r ALL and hope to build on these findings as they advance the CTL019 clinical program in Phase II trials.

Novartis recently established the Cell and Gene Therapies Unit to focus on advancing cell-based therapies, including the development of CARs. Novartis holds the worldwide rights to CARs developed through the collaboration with Penn for all cancer indications, including the lead program, CTL019.