Novartis on Sunday announced the long-term results from the ELIANA pivotal clinical trial of Kymriah® (tisagenlecleucel), the first-ever approved CAR-T cell therapy, in children and young adult patients with relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (ALL), with a maximum survival follow-up of 5.9 years.
According to the company’s press statement, for the 79 patients treated with Kymriah in this study, the five-year overall survival (OS) rate was 55 percent (95% CI, 43-66), while the median event-free survival (EFS) for patients in remission within three months of infusion (n=65) was 43.8 months.
“These findings demonstrate the curative potential of Kymriah, the only CAR-T cell therapy available for these patients who previously had limited treatment options. These data were presented as an oral presentation during the 2022 European Hematology Association (EHA) Hybrid Congress (Abstract #S112),” the company said in its press statement.
“These data mark a moment of profound hope for children, young adults, and their families with relapsed or refractory B-cell ALL, as relapse after five years is rare. Since the approval of Kymriah nearly five years ago, we have been able to offer a truly game-changing option to patients who previously faced a five-year survival rate of less than 10 percent,” said Stephan Grupp, MD, PhD, Section Chief of the Cellular Therapy and Transplant Section, and Inaugural Director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy at Children’s Hospital of Philadelphia (CHOP).
The company claims that this long-term follow-up of ELIANA demonstrated the potential for Kymriah to transform cancer treatment in pediatric and young adult patients with r/r B-cell ALL, significantly improving outcomes with durable responses and consistent safety profile in this patient population1.
“At Novartis, we strive for cures. With nearly six-year follow-up data in these pediatric and young adults treated for B-cell ALL, we have our strongest evidence yet that one-time treatment with Kymriah has curative potential. These results strengthen our confidence in CAR-T cell therapies as a truly transformative and paradigm-shifting advance in cancer care, as well as our commitment to continue developing this technology with next-generation platforms,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development.