FDA Awards Breakthrough Status To Penn And Novartis' Immunotherapy

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy status to CTL019, an immunotherapy developed by the University of Pennsylvania and licensed to Novartis, for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia (ALL).

CTL019 is an investigational chimeric antigen receptor (CAR) therapy designed as a personalized cellular therapy for the treatment of cancer. CAR technology, in essence, reprograms T cells to target cancer cells that express the protein CD19. The reengineered CTL019 T cells are reintroduced to the patient’s blood stream where they multiply and bind to the hunted CD19+ cancer cells to destroy them.

The FDA assigned Breakthrough status to CTL019 based on positive clinical trial data showing that 89 percent of ALL patients unresponsive to current therapies entered complete remission after receiving CTL019 treatment.

“Our early findings reveal tremendous promise for a desperate group of patients, many of whom have been able to return to their normal lives at school and work after receiving this new, personalized immunotherapy,” said the Penn research team leader Carl June, director of Translational Research in the Abramson Cancer Center of the University of Pennsylvania.

David Epstein, Division Head, Novartis Pharmaceuticals, said, “This Breakthrough Therapy designation underscores the potential of CTL019 as a life-saving therapy for patients with relapsed/refractory ALL, who are in desperate need of new treatment options. Novartis welcomes increased dialogue with the FDA and a potentially expedited review to streamline the development of CTL019 and hopefully bring this promising therapy to patients as quickly as possible.”

Acute lymphoblastic leukemia (ALL) is the leading cancer diagnosed in children, with an estimated 25 percent of diagnoses in patients younger than 15 years of age—though the disease can occur in both children and adults. ALL is characterized by the production of abnormal white blood cells.

Penn signed an exclusive global research and licensing agreement with Novartis in August 2012 for the development and marketing of CAR T cell therapies. Trials are ongoing for ALL patients as well as those with non-Hodgkin lymphoma and myeloma. The partners are also investigating next generation CAR therapies in trials for early stage pancreatic cancer, ovarian, breast, and mesothelioma. This partnership would also result in the construction of a new Center for Advanced Cellular Therapies (CACT) on Penn’s campus where researchers will work to discover, develop, and manufacture T-Cell immunotherapies.