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# FDA Approves Neuroblastoma Drug
- URL: https://www.fdaweb.com/fda-approves-neuroblastoma-drug/
- Published: 2023-12-14T12:00:00.000Z
- Updated: 2026-09-14T14:18:36.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5156003

FDA has approved a U.S. WorldMeds NDA for eflornithine for reducing the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy.

Approval was based on data from two clinical trials (Study 3b and Study ANBL0032). The major efficacy outcome measures were event free survival (EFS) and overall survival (OS). “In the protocol-specified primary analysis, the EFS hazard ratio (HR) was 0.48 (95% CI: 0.27, 0.85) and OS HR was 0.32 (95% CI: 0.15, 0.70),” an [agency release](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-eflornithine-adult-and-pediatric-patients-high-risk-neuroblastoma?ref=fdaweb.com) says. “Given the uncertainty in treatment effect estimation associated with the externally controlled study design, supplementary analyses in subpopulations or using alternative statistical methods were performed. In these analyses, the EFS HR ranged from 0.43 (95% CI: 0.23, 0.79) to 0.59 (95% CI: 0.28, 1.27), and the OS HR ranged from 0.29 (95% CI: 0.11, 0.72) to 0.45 (95% CI: 0.21, 0.98).”

Regarding safety, the most common adverse reactions, including laboratory abnormalities, were “otitis media, diarrhea, cough, 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,” FDA says.