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Novartis five-year Kymriah data show durable remission and long-term survival maintained in children and young adults with advanced B-cell ALL.- Novartis

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Published:13th Jun 2022

Novartis announced long-term five-year follow-up 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) .


For the 79 patients treated with Kymriah in this study, the five-year overall survival (OS) rate was 55% (95% CI, 43-66) and the median event-free survival (EFS) for patients in remission within three months of infusion (n=65) was 43.8 months. The five-year EFS without censoring for alloSCT: 36% (95% CI, 25%-47%) The five-year EFS with censoring for alloSCT: 34% (95% CI, 23%-45%). 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 #3782).

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 a consistent safety profile in this patient population: • Eighty-two percent of patients experienced remission (either complete remission [CR] or CR with incomplete hematologic recovery within three months after infusion) (95% CI, 72-90). • For patients in remission, Five-year relapse-free survival (RFS) rate was 44% (95% CI, 31-56) and the median RFS was 43 months. • No new or unexpected adverse events were reported during long-term follow-up.

“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,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development. “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.”

Additional updates on the Novartis CAR-T program presented at the 2022 EHA Congress include new data from more patients and longer follow-up from the first-in-human dose-escalation trials with YTB323 in adults with r/r diffuse large B-cell lymphoma and PHE885 in adults with r/r multiple myeloma, the first Novartis CAR-T cell therapies developed using the Novartis T-Charge™ platform.

About the ELIANA Trial : ELIANA was the first pediatric global CAR-T cell therapy registration trial, examining patients in 25 centers in 11 countries across the US, Canada, Australia, Japan and the EU, including: Austria, Belgium, France, Germany, Italy, Norway and Spain. The trial was an open-label, multicenter, single-arm, global Phase II trial investigating the efficacy and safety of Kymriah in pediatric and young adult patr, single-arm, global Phase II trial investigating the efficacy and safety of Kymriah in pediatric and young adult patients in r/r B-cell ALL who were primary refractory, chemorefractory, relapsed after, or were not eligible for allogeneic stem cell transplantation (SCT). The primary endpoint was overall remission rate (ORR), defined as best overall response of CR or CR with incomplete blood count recovery (CRi) within 3 months and maintained for ?28 day. The secondary endpoints include CR/CRi with undetectable minimal residual disease (MRD), duration of remission, event-free survival, overall survival, cellular kinetics and safety.

Condition: B-Cell Acute Lymphoblastic Leukemia
Type: drug

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