Dice Novartis tener la autorización de la Comisión Europea para Kymriah, aunque en la página de la Ema, a día de hoy, aún aparece como pendiente.
The EC approval is based on the first global CAR-T registration trials, which included patients from eight European countries and demonstrated durable responses and a consistent safety profile in r/r pediatric B-cell ALL and r/r DLBCL.
Novartis is the only company with an approved CAR-T cell therapy for pediatric r/r B-cell ALL and the first to receive approval in two distinct indications, both in the EU and the US
El informe favorable estaría basado en los ensayos clínicos ELIANA para la recidiva de LLA en menores de 25 años:
The EC approval of Kymriah in pediatric and young adult patients with r/r B-cell ALL is based on the pivotal Phase II ELIANA clinical trial, the first pediatric global CAR-T cell therapy registration study for Kymriah in children and young adults with r/r B-cell ALL. ELIANA was conducted in collaboration with the University of Pennsylvania and Children’s Hospital of Philadelphia, evaluating Kymriah in patients in 25 centers in the US, Canada, Australia, Japan, and in Europe, in Austria, Belgium, France, Germany, Italy, Norway and Spain.
In this Novartis-sponsored, global, multi-center study evaluating 75 patients infused with Kymriah with three or more months of follow-up, 81% of patients achieved overall remission (95% CI: 71% - 89%) with 80% of responders still in remission at 6 months. Sixty percent of patients achieved complete response (CR) and 21% of patients achieved CR with incomplete blood count recovery (CRi). Of those patients in remission, 100% had no minimal residual disease (MRD) detected in the bone marrow1. Overall survival (OS) was 90% at six months, and 76% at 12 months. Median OS was 19.1 months (95% CI: 15.2 - NE) in this difficult-to-treat patient population.
y JULIET para los linfomas difusos de células B grandes en adultos:
The EC approval of Kymriah in adult patients with r/r DLBCL is based on the pivotal Phase II JULIET clinical trial, the first multi-center global registration study for Kymriah in adult patients with r/r DLBCL. JULIET was conducted in collaboration with the University of Pennsylvania, and is the largest study examining a CAR-T therapy in DLBCL, enrolling patients from 27 sites in 10 countries across the US, Canada, Australia, Japan, and Europe in Austria, France, Germany, Italy, Norway and the Netherlands.
In the JULIET trial, patients were infused in the inpatient and outpatient setting. In this Novartis-sponsored, global, multi-center study, among 93 evaluable patients who were followed for at least three months or discontinued earlier, Kymriah demonstrated an overall response rate (ORR) of 52% (95% confidence interval [CI], 41% - 62%), with 40% achieving a complete response (CR) and 12% achieving a partial response (PR). The relapse-free probability at 6 and 12 months was 68% and 65%, respectively; and the median duration of response was not reached at the time of data cut-off, indicating sustainability of response1. The OS rate at 12 months was 49% and median OS was 11.7 months among all infused patients (n=111) (95% CI, 6.6-NE).
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