Updated results from the pivotal ELIANA clinical trial of Novartis’ KymriahTM (tisagenlecleucel), formerly CTL019, in relapsed or refractory (r/r) paediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL) have been published in The New England Journal of Medicine.
New data include longer-term follow-up and efficacy in 75 infused patients, analysis of expansion and persistence of Kymriah, and longer-term safety. Kymriah became the first chimeric antigen receptor T (CAR-T) cell therapy to receive regulatory approval in August 2017, when it was approved by the US Food and Drug Administration (FDA) for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse, based on previous results from the ELIANA study, which was conducted in collaboration with the University of Pennsylvania (Penn) and the Children’s Hospital of Philadelphia.
In the analysis of 75 infused patients with three or more months of follow-up, Kymriah demonstrated an overall remission rate of 81% (95% CI: 71% – 89%). 60% of patients achieved complete remission (CR) and 21% of patients achieved CR with incomplete blood count recovery (CRi), with no minimal residual disease detected among all responding patients (95% [58/61] by day 28). Median follow-up was 13.1 months.
ELIANA is the first paediatric global CAR-T cell therapy registration trial, examining patients in 25 centres in the US, Canada, Australia, Japan and the EU, including Austria, Belgium, France, Germany, Italy, Norway and Spain, demonstrating effective distribution of CTL019 across four continents using a global supply chain.
In 2012, Novartis and Penn entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including Kymriah, for the investigational treatment of cancers.
Dr Samit Hirawat, Head, Novartis Oncology Global Drug Development, said: “These data are a testament to our commitment at Novartis for continued CAR-T cell therapy research to bring this therapy to as many patients as possible.”
Lead study author Dr Shannon L. Maude, Assistant Professor of Peadiatrics, at Children’s Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, said: “We continue to be encouraged by the results demonstrated with Kymriah in a patient population who previously had limited treatment options, and now have the potential for durable remissions translating into longer-term survival.”
A Marketing Authorisation Application for Kymriah for the treatment of children and young adults with r/r B-cell ALL and adult patients with r/r diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant is currently under review by the European Medicines Agency (EMA).