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FDA Approves Eflornithine for High-Risk Neuroblastoma to Reduce Relapse Risk

• The FDA has approved eflornithine (Iwilfin) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB). • The approval is for patients who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy. • Efficacy was demonstrated in a controlled trial comparing eflornithine to an external control arm, showing improved event-free and overall survival. • Common adverse events in the eflornithine arm included otitis media, diarrhea, cough, and various infections, alongside some lab abnormalities.

The FDA has granted approval to eflornithine (Iwilfin) for reducing the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB). This approval is specifically for patients who have achieved at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy. The decision marks a significant advancement in the treatment landscape for this challenging pediatric cancer.
The approval was based on data from a controlled trial that compared outcomes from Study 3b (the eflornithine arm) and Study ANBL0032 (a clinical trial-derived external control arm). In October 2023, the FDA's Oncologic Drug Advisory Committee (ODAC) voted 14 to 6 in favor of eflornithine, citing sufficient evidence of its ability to reduce relapse risk in HRNB patients who are in remission and have completed multi-agent, multi-modality therapy.

Study Design and Efficacy

Study 3b was a multi-center, open-label, non-randomized trial involving two cohorts. The analysis focused on 105 eligible patients with HRNB who received oral eflornithine twice daily, with the dosage adjusted based on body surface area. Treatment continued until disease progression, unacceptable toxicity, or a maximum of two years.
The external control arm consisted of 1,241 patients from Study ANBL0032, a multi-center, open-label, randomized trial evaluating dinutuximab, granulocyte-macrophage colony-stimulating factor, interleukin-2, and cis-retinoic acid compared to cis-retinoic acid alone in pediatric patients with HRNB. Propensity score matching was used to create comparable groups, resulting in 90 patients in the eflornithine arm and 270 control patients from ANBL0032 for the primary analysis.
The primary endpoint was event-free survival (EFS), defined as disease progression, relapse, secondary cancer, or death due to any cause. Overall survival (OS), defined as death due to any cause, was a key secondary endpoint. The hazard ratio (HR) for EFS was 0.48 (95% CI, 0.27-0.85), and the HR for OS was 0.32 (95% CI, 0.15-0.70). Additional analyses showed EFS HRs ranging from 0.43 (95% CI, 0.23-0.79) to 0.59 (95% CI, 0.28-1.27) and OS HRs from 0.29 (95% CI, 0.11-0.72) to 0.45 (95% CI, 0.21-0.98).

Safety Profile

The most common adverse events observed in ≥5% of patients in Study 3b included otitis media, diarrhea, cough, sinusitis, pneumonia, upper respiratory tract infection, conjunctivitis, vomiting, pyrexia, allergic rhinitis, decreased neutrophils, increased alanine transaminase, increased aspartate transaminase, hearing loss, skin infection, and urinary tract infection. Healthcare providers should monitor patients for these potential side effects during eflornithine treatment.
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Eflornithine Approved by FDA for High-Risk Neuroblastoma to Reduce Relapse Risk

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Reference News

[1]
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targetedonc.com · Dec 13, 2023

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[2]
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cancernetwork.com · Dec 13, 2023

FDA approved eflornithine (Vaniqa) for high-risk neuroblastoma patients showing partial response to prior treatment. Stu...

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