Novartis has submitted an application to the FDA to extend the indications for its CAR T-cell therapy tisagenlecleucel (Kymriah) for its use in adult patients with relapsed or refractory diffuse large B-cell lymphoma who are not eligible for autologous stem cell transplant.
The pharmaceutical company Novartis has submitted an application to the US Food and Drug Administration (FDA) to extend the indications for its chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel (Kymriah). The application is for its use in adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant.
“The data show this therapy could change the treatment paradigm for patients with relapsed/refractory DLBCL,” said Stephen Schuster, MD, of the University of Pennsylvania Perelman School of Medicine in Philadelphia, according to a press release.
CAR T-cell therapies have started to gain approvals in recent months. Tisagenlecleucel was first approved at the end of August for treatment of pediatric and young adult patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (ALL), and in October the FDA approved axicabtagene ciloleucel (Yescarta) for use in patients with de novo and transformed large B-cell lymphoma and high grade B-cell lymphoma in adults who failed or relapsed following two or more prior therapies.
The new application for use in DLBCL is based on results of the phase II JULIET trial, a multi-center trial being conducted in 10 countries including the United States, Canada, Japan, Australia, and in Europe. The primary analysis results of the trial will be presented at the American Society of Hematology annual meeting, to be held December 9–12 in Atlanta.
“We’ve seen durable complete responses in patients who previously relapsed or were refractory to prior therapies, and this second filing is a significant step toward realizing its potential for even more patients who are currently battling fatal blood cancers,” Schuster said. The global head of drug development and chief medical officer for Novartis, Vas Narasimhan, added that the good response rates seen in the JULIET trial show the therapy “has the potential to transform treatment for these patients.”
CAR T-cell therapy involves tailoring the agent specifically to each patient. T cells are drawn from the patient, and then genetically modified to specifically target cancer cells. In other trials involving tisagenlecleucel, cytokine release syndrome (CRS) was a significant adverse effect; its previous approval included a risk evaluation and mitigation strategy to mitigate the risks of CRS and other neurologic toxicities.