Novartis has shared long-term, five-year follow-up results from its pivotal clinical phase 2 trial, ELIANA, evaluating 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 leukaemia (ALL).
Kymriah is a one-off treatment designed to help patients’ immune systems to fight cancer and is the first CAR T-cell therapy to receive approval from the US Food and Drug Administration (FDA).
It is currently approved to treat lymphoblastic leukaemia in paediatric cases and in young adults 25 years and younger, as well as to treat diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma in adults.
In the ELIANA study, for the 79 patients treated with Kymriah, the five-year relapse-free survival (RFS) rate was 44% and the median RFS was 43 months the five-year, while the overall survival rate was 55%. The safety profile reported in the trial remains consistent with previous results.
Jeff Legos, executive vice president, Novartis, global head of oncology and haematology development, commented: “With nearly six-year follow-up data in these paediatric and young adults treated for B-cell ALL, we have our strongest evidence yet that one-time treatment with Kymriah has curative potential,”
“The 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.”
The data shows the potential of Kymriah as a cure, and as the only CAR T-cell therapy available for such patients who have previously had limited treatment options.
The data taken from the trial was presented during the 2022 European Hematology Association (EHA) Hybrid Congress.
“This data marks 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,” said Stephan Grupp, 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).
He added: “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%.”
The ELIANA trial was the first worldwide CAR T-cell therapy registration study, assessing patients in 25 centres in 11 countries including the EU, the US, Canada, Australia and Japan.