The FDA’s Oncologic Drugs Advisory Committee on Wednesday voted 14 to 6 in favor of the efficacy of US WorldMeds’ potential therapy to reduce the risk of relapse in pediatric patients with high-risk neuroblastoma.
The vote sets up a tricky and potentially precedent-setting decision for FDA, as adcomm members questioned both FDA and the sponsor over why a randomized clinical trial (RCT) for the therapy, known as eflornithine or DFMO, was not run. FDA recommended an RCT back in 2015, but US WorldMed insisted that its externally controlled trial results are enough to support the use of eflornithine in this hard-to-treat population of those who have already received other therapies.
While calling this meeting “a fairly extraordinary situation,” ODAC chair Christopher Lieu, director of the gastrointestinal medical oncology program at the University of Colorado, voted “yes,” and summarized the committee’s discussion by noting a “sincere appreciation” for the effort that…
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