FDA Grants Breakthrough Therapy Designation to Investigational Chimeric Antigen Receptor Therapy for Relapsed/Refractory ALL


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The U.S. 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 (ALL). The Breakthrough Therapy filing was submitted by the University of Pennsylvania’s Perelman School of Medicine which has an exclusive global agreement with Novartis to research, develop, and commercialize personalized CAR T-cell therapies for the treatment of cancers.

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.

About CTL019

CTL019 uses CAR technology to reprogram a patient’s own T cells to target CD19-expressing cancer cells. After they have been reprogrammed, the T cells (now called CTL019) are reintroduced into the patient’s blood where they proliferate and bind to the targeted CD19-positive cancer cells and destroy them. Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established.

“This is a major milestone as we are now one step closer in helping address the high unmet needs of this patient population,” said Carl H. June, MD, Richard W. Vague Professor in Immunotherapy in the Department of Pathology and Laboratory Medicine in the Perelman School of Medicine and Director of Translational Research in the Abramson Cancer Center of the University of Pennsylvania. “We are excited about the strength of the positive early data seen in pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia and look forward to building upon these findings as we continue advancing the CTL019 clinical program in phase II trials.” ■



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