Novartis’ Kymriah has become the first ever CAR-T cell therapy to win approval in the US, bringing paediatric and young adult patients with B-cell acute lymphoblastic leukaemia a novel treatment approach.
In a historic decision, the US Food and Drug Administration is allowing use of Kymriah (tisagenlecleucel; CTL019) for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukaemia (ALL) that is refractory or in second or later relapse.
CAR-T offers a new treatment approach in that it is specifically manufactured for each individual patient. During the process, T cells are drawn from a patient's blood and reprogrammed in the lab to create T cells that are genetically coded to hunt the patient's cancer cells.
Novartis says its first-in-class therapy showed an 83 percent overall remission rate in this patient population with limited treatment options and historically poor outcomes.
"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,” said Bruno Strigini, chief executive of Novartis Oncology.
The firm also highlighted the “urgent need” for new treatment options that boost outcomes for patients with relapsed or refractory (r/r) B-cell precursor ALL, whose prognosis is poor. Patients often undergo multiple treatments including chemotherapy, radiation, targeted therapy or stem cell transplant, yet less than 10 percent are alive after five years.
"Tisagenlecleucel is the first CAR-T therapy to demonstrate early, deep and durable remission in children and young adults with relapsed or refractory B-cell ALL," said Stephan Grupp, the Yetta Deitch Novotny Professor of Pediatrics at the Perelman School of Medicine at Penn, and director of the Cancer Immunotherapy Frontier Program at Children's Hospital of Philadelphia. "We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population."
Novartis picked up rights to Kymriah under an agreement with the University of Pennsylvania in 2012, which also gives it worldwide rights to CAR-Ts developed in all cancer indications.
The decision is also great news for UK drugmaker Oxford BioMedica, which is the sole manufacturer of the lentiviral vector that encodes Kymriah.
Under the companies’ deal, sealed in July 2017, Oxford BioMedica also will supply lentiviral vectors for other undisclosed CAR-T products, and could potentially receive in excess of $100 million from Novartis over the next three years in return, as well as royalties on future sales.
“We are delighted that the FDA has now approved CTL019,” said John Dawson, Oxford BioMedica’s chief executive. “This innovative immunocellular therapy will offer critically ill patients with an aggressive disease a new treatment option.”
Eyes will now be on Kite Pharma’s CAR-T axicabtagene ciloleucel (axi-cel), which is currently under priority review by the US Food and Drug Administration as a treatment for refractory aggressive non-Hodgkin lymphoma (NHL), a decision for which is expected sometime in the fourth quarter.