Novartis is a step closer to the U.S. Food and Drug Administration approving its CAR T-cell therapy CTL019 for certain patients with leukemia.
The FDA has accepted Novartis’ Biologics License Application for CTL109. The application specifies that the therapy be used to treat children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia.
The good news includes the fact that the FDA granted priority review to Novartis’ application. That will shorten the time it takes for the agency to make a decision from the usual 10 months to six.
“With CTL019, Novartis is at the forefront of the science and development of immunocellular therapy as a potential new innovative approach to treating certain cancers where there are limited options,” Vas Narasimhan, Novartis’ chief medical officer, said in a press release. “The priority review and file acceptance of CTL019 by the FDA brings us one step closer to delivering this novel treatment option to children and young adults with r/r B-cell ALL in the United States.”
CAR T-cells are T-cells genetically engineered to recognize and attack a particular tumor. They are harvested from a patient and taken to a lab, where researchers modify them to target a specific cancer protein.
Scientists then expand the cells, or generate millions that can recognize the protein. They are then injected back into the patient to attack cancer cells.
The University of Pennsylvania developed CTL109 to recognize the CD19 protein, which is widely expressed in lymphoma and leukemia cells.
The FDA’s decision to accept Novatis’ application, and accelerate the review process, was based on results of the Phase 2 ELIANA study (NCT02435849), the first global clinical trial of a CAR T-cell therapy.
The study enrolled 50 children and young adult patients with relapsed or refractory B-cell ALL at 25 centers in the United States, Europe, Canada, Australia, and Japan. Participants received a single infusion of CTL019.
Data presented at the American Society of Hematology meeting in December 2016 showed that three months after the infusion, 82 percent of patients had achieved either a complete remission, or a complete remission with an incomplete blood count recovery.
Nearly half of the patients experienced grade 3 or 4 cytokine release syndrome (CRS), an immunotherapy complication caused by immune system over-activation. Doctors managed CRS according to a predefined protocol, however, and no patients died due to the side effect.
Novartis included in its application the results of a single-site study at the Children’s Hospital of Philadelphia. The study, led by Stephan Grupp, MD, PhD, also examined the safety and effectiveness of CTL019 in children and young adults with relapsed or refractory B-cell ALL.
“Even if a patient has difficult-to-treat relapsed/refractory leukemia, we have seen treatment with CTL019 in clinical trials put cancer into remission,” said Grupp, director of translational research at the hospital’s Center for Childhood Cancer Research. “This could be a first-of-its-kind treatment with exciting potential to help pediatric and young adult r/r B-cell ALL patients.”
Novartis plans additional marketing applications for CTL019 later this year. In the United States it will ask that the therapy be approved to treat adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). In Europe it will ask that CTL109 be approved to treat relapsed or refractory B-cell ALL and DLBCL.
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