FDA Approves Eflornithine for Pediatric Neuroblastomas

The FDA's Oncologic Drug Advisory Committee (ODAC) has voted in favor of approving eflornithine (DFMO) for reducing the risk of relapse in pediatric patients with high-risk neuroblastoma (HRBN) who are in remission and have completed multi-agent, multi-modality therapy. This decision is significant as it marks the first time the FDA has relied on a single externally controlled trial to support a potential approval in oncology. The trial, known as study 3b, presented external control data from a previous study and showed promising results in reducing relapse rates. The FDA's decision was influenced by the high unmet medical need for neuroblastoma patients and the availability of the external control data.