FDA Panel Recommends Approval for Novartis CAR-T Cell Therapy

A breakthrough cancer treatment is one step closer to regulatory approval.

The U.S. Food and Drug Administration’s Oncologic Drugs Advisory Committee unanimously voted to recommend approval for CTL019, an experimental cancer drug that belongs to a class of drugs called CAR T-cell therapies, which is a form of immunotherapy that essentially takes control of the body’s T-cells and directs them to attack tumors.

Gaining approval for CTL019 could make it the first officially approved gene therapy in the United States.

“The panel’s unanimous recommendation in favor of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need,” explained Novartis Oncology CEO Bruno Strigini, in a statement. We’re very proud to be expanding new frontiers in cancer treatment by advancing immunocellular therapy for children and young adults with r/r B-cell ALL and other critically ill patients who have limited options. We look forward to working with the FDA as they complete their review.”

Novartis is developing the drug to treat either relapsed or refractory pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). This type of cancer comprises approximately 25 percent of cancer diagnoses with children under 15 years old and serves as the most common childhood cancer in the United States.

The FDA panel made their recommendation after reviewing CTL019’s performance in a range of clinical trials including the ELIANA study, which was the first pediatric global CAR-T cell therapy registration trial.

Also, results from a trial focusing on the most common form of childhood cancer demonstrated that 82 percent of patients that had relapsed or failed chemotherapy produced a complete response rate within one to three months of treatment.

The notable aspect of these findings was that patients with ALL who failed chemotherapy usually only have a 16 to 30 percent chance of survival, noted Reuters.

“It is encouraging to see the FDA panel’s recommendation and continued momentum behind this innovative therapy, which has potential to help young patients with relapsed/refractory B-cell ALL,” said the Dr. Carl June, MD, the Richard W. Vague Professor of Immunotherapy, director of the Center for Cellular Immunotherapies in Penn’s Perelman School of Medicine and director of the Parker Institute for Cancer Immunotherapy at Penn, who helped work on developing this drug. “We look forward to continuing to work with Novartis to help make a lasting impact on the way this disease is treated.

Next, Novartis plans on making additional filings for the drug in the US and EU later this year.

The FDA does not have to follow the recommendations of these panels, but typically does so, with the expectation the agency could make a decision on the drug by the end of September.