Trial Design

The efficacy of IWILFIN is based on an externally controlled trial comparison of outcomes between Study 3b (investigational arm) and Study ANBL0032 (clinical trial–derived external control arm). 

  • Study 3b (IWILFIN-treated patients): A multicenter, open-label, non-randomized trial. Pediatric patients with high-risk neuroblastoma who demonstrated at least a partial response to the standard of care upfront therapy, consistent with ANBL0032 treatment protocol, received IWILFIN orally twice daily for a maximum of 2 years 
  • Study ANBL0032 (external control): A multicenter, open-label, randomized trial of dinutuximab, granulocyte-macrophage colony-stimulating factor, interleukin-2, and cis-retinoic acid compared to cis-retinoic acid alone in pediatric patients with high-risk neuroblastoma 

Patients who met the criteria for the comparison and had complete data for specified clinical covariates were matched (1:3) using propensity scores; the matched efficacy populations for the primary analysis included 90 patients treated with IWILFIN and 270 control patients from ANBL0032.

ASCT, autologous stem cell transplantation; CR, complete response; HRNB, high-risk neuroblastoma; INSS, International Neuroblastoma Staging System; PR, partial response; VGPR, very good partial response. 

IWILFIN improved outcomes in primary matched comparison1,2  

Primary Endpoint: Event free survival (EFS)

EFS is defined as the period from the last day of immunotherapy until the first occurrence of relapse, progressive disease, secondary cancer, or death.

Secondary Endpoint: Overall survival (OS)

OS is defined as the first day of administration of IWILFIN until death due to any cause.

Of 360 patients with high-risk neuroblastoma who received IWILFIN as maintenance therapy in clinical studies, IWILFIN was generally well tolerated 

ALT, alanine aminotransferase; AST, aspartate aminotransferase. 

Based on the severity of adverse reactions to IWILFIN, reduce the patient’s dose or stop treatment until the reaction subsides or resolves to baseline1. Please see recommended dose reductions contained in the Prescribing Information. 


1. IWILFIN. Prescribing information. USWM, LLC; 2023

2. Data on file. USWM, LLC.