Novartis Gains FDA Breakthrough Therapy Status for Cancer Drug
Novartis reports that the United States Food and Drug Administration (FDA) has 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.
CTL019 uses CAR technology to reprogram a patient’s own T cells to “hunt” cancer cells that express specific proteins, called CD19. After they have been reprogrammed, the T cells (now called CTL019) are re-introduced into the patient’s blood; they proliferate and bind to the targeted CD19+ cancer cells and destroy them.
Novartis recently established the Cell and Gene Therapies Unit under the leadership of Usman Azam, global head, to focus on advancing innovative 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.
This is the fifth Breakthrough Therapy designation for Novartis. Novartis’ Zykadia(TM) (ceritinib) for the treatment of anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer (NSCLC), was one of the first medicines that received Breakthrough Therapy status to receive FDA approval.
According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of new medicines that treat serious or life-threatening conditions if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint. The designation includes all of the fast track program features as well as more intensive FDA guidance. It is a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.