FDA approves eflornithine for pediatric patients with high-risk neuroblastoma

FDA approves eflornithine for pediatric patients with high-risk neuroblastoma | Image Credit: © calin - © calin - stock.adobe.com.

The FDA has approved eflornithine (IWILFIN; US WorldMeds) 192 mg tablets to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have had at least a partial response to prior multiagent, multimodality therapy.^1,2

According to the federal agency, the approval of eflornithine is the first for a therapy intended to reduce relapse for pediatric patients with HRNB.¹

Each year in the United States, there are approximately 700 to 800 cases of NB, of which 90% are diagnosed before the age of 5 years. More than half of these cases are considered high-risk.²

According to US WorldMeds, approximately half of children with HRNB do not survive past 5 years from diagnosis. Avoiding relapse is crucial to improve survival rates.² 

The approval of eflornithine was based on results from an externally controlled trial comparing outcomes from Study 3b (NCT02395666) and Study ANBL0032, a clinical trial-derived external control arm.¹

Study 3b was a multi-center, open label, non-randomized trial with 2 cohorts. Eligible patients in one cohort received eflornithine orally, twice daily at a dosage based on body surface area until disease progression, unacceptable toxicity, or for a maximum of 2 years, according to the FDA.¹

The study was designed to compare outcomes to the historical benchmark event free survival (EFS) rate from Study ANBL0032, a multi-center, 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 HRNB.”¹

Eligible patients for the comparative analysis of Study 3b and ANBL0032 were matched 1 to 3 using propensity scores. Ninety patients treated with eflornithine and 270 control patients from Study ANBL0032 were the matched efficacy populations for the primary analysis.¹

EFS, defined as disease progression, relapse, secondary cancer, or death due to any cause, was the major efficacy outcome of the analysis. Overall survival (OS) (death because of any cause) was a secondary efficacy outcome.¹

Results demonstrated that the addition of eflornithine improved EFS and OS for patients with HRNB. At 4 years post immunotherapy, EFS in eflornithine-treated patients was 84% compared to 73% of patients in the external control group.²

Additionally, 96% of eflornithine-treated patients were alive compared to 84% of control patients, which corresponded to a 52% reduction in the risk of relapse and a 68% reduction in the risk of death.²

Otitis media, cough, diarrhea, sinusitis, pneumonia, upper respiratory tract infection, conjunctivitis, vomiting, pyrexia, allergic rhinitis, decreased neutrophils, increased ALT, increased AST, hearing loss, skin infection, and urinary tract infection were the most common adverse reactions (≥5%).¹

The application for eflornithine was granted Priority Review, Breakthrough, and Orphan Drug designations by the FDA.¹

References:

1. FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma. FDA. Press release. December 13, 2023. Accessed December 14, 2023. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-eflornithine-adult-and-pediatric-patients-high-risk-neuroblastoma?utm_medium=email&utm_source=govdelivery
2. US WorldMeds announces FDA approval of IWILFIN (eflornithine) to strengthen fight against aggressive childhood cancer. US WorldMeds. Press release. December 14, 2023. Accessed December 14, 2023.