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US FDA’s Eflornithine Review Is Showcase For Deep Oncology Team

Executive Summary

The advisory committee review of US WorldMeds’ eflornithine for neuroblastoma was remarkable for the unusual nature of the efficacy data to support the NDA – and also for who spoke on behalf of the agency and how effectively they walked the panel through a complex set of issues.

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Animal Models: Adcomm Exposes Internal Rift In How FDA Defines ‘Translational’ For Purposes Of Confirmatory Evidence

FDA review staff consider the animal models used in development of US WorldMeds’ eflornithine for neuroblastoma to be translational to humans, even though this does not align with the definition in a September 2023 draft guidance on types of confirmatory evidence.

US WorldMeds’ Neuroblastoma Drug: External Controls, Confirmatory Evidence, And A Concern About Precedent

US FDA officials said the high-quality nature of the patient-level data used in the external control arm, and the use of animal models considered to be translatable, justify reliance upon a nontraditional data package for eflornithine (DFMO) in high-risk neuroblastoma, but some adcomm members worry that an approval will open the door too widely for others to follow.

US FDA Eyes Ways To Improve Conduct, Mitigate Bias In Open-Label Cancer Studies

At advisory committee review of confirmatory trial results for Amgen’s Lumakras, Oncology Center of Excellence’s Richard Pazdur cites need for a broader conversation within the clinical trial community, including educating patients and investigators about the importance of staying on study even when randomized to a comparator arm.

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