Novartis has received a FDA approval for its CAR-T treatment tisagenlecleucel, a first-ever cell therapy for a rare form of acute lymphoblastic leukemia.
The US Food and Drug Administration (FDA) has approved Kymriah (tisagenlecleucel) suspension for intravenous infusion, formerly CTL019, the first chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. Kymriah is a novel immunocellular therapy and a one-time treatment that uses a patient’s own T cells to fight cancer. Kymriah is the first therapy based on gene transfer approved by the FDA.
The road to approval for CAR-T has been filled with uncertainty, but Novartis and lead rival Kite Pharma made strides over the last year, notably with Novartis’ recent unanimous FDA panel vote in favor of approval and Kite’s $11.9 billion acquisition this week by Gilead Sciences, reports FiercePharma. The Novartis medication is made of re-engineered T cells collected from each patient, which are then infused back into the patient to attack cancer. It will be targeted at children and young adult patients. In the agency’s approval announcement, new FDA commissioner Scott Gottlieb said the medical community is “entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer.”
Twenty treatment centers will support the launch initially, with the number growing to 32 by the end of the year, execs said Wednesday. Some centers will begin harvesting patients’ T cells in the next 3 to 5 days. The company has been “working with payers for a long time,” Novartis’ U.S. oncology head Bill Hinshaw said on the call, adding that the company anticipates “excellent coverage” based on those discussions.
“We are so proud to be part of this historic moment in cancer treatment and are deeply grateful to our researchers, collaborators, and the patients and families who participated in the Kymriah clinical program,” said Bruno Strigini, CEO of Novartis Oncology. “As a breakthrough immunocellular therapy for children and young adults who desperately need new options, Kymriah truly embodies our mission to discover new ways to improve patient outcomes and the way cancer is treated.”
The FDA has approved a Risk Evaluation and Mitigation Strategy (REMS) for Kymriah. The REMS program serves to inform and educate healthcare professionals about the risks that may be associated with Kymriah treatment. To support safe patient access, Novartis is establishing a network of certified treatment centers throughout the country which will be fully trained on the use of Kymriah and appropriate patient care.
Novartis plans additional filings for Kymriah in the US and EU later this year, including applications with the FDA and European Medicines Agency (EMA), for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL). Additional filings beyond the US and EU are anticipated in 2018.