Members of the FDA’s Oncologic Drugs Advisory Committee indicated there isn’t enough evidence to definitively confirm the overall survival benefit of 131I-omburtamab in pediatric patients with central nervous system or leptomeningeal metastases stemming from neuroblastoma.
A 16-to-0 vote was cast by the FDA’s Oncologic Drugs Advisory Committee (ODAC) against 131I-omburtamab as a treatment for pediatric patients with central nervous system (CNS) or leptomeningeal metastases from neuroblastoma, according to a press release from Y-mAbs.1
After reviewing findings from the phase 1 study 03-133 (NCT00089245)—assessing 131I-omburtamab in patients with refractory, recurrent, or advanced CNS or leptomeningeal cancer—and the phase 1/2 study 101 (NCT03275402)—assessing the agent in neuroblastoma with CNS or leptomeningeal metastases—the ODAC concluded that there was not sufficient evidence to confirm the overall survival (OS) benefit of 131I-omburtamab.
“We are disappointed by the outcome of today’s meeting, as patients with CNS/leptomeningeal metastasis from neuroblastoma are in need of effective and safe treatment options,” Thomas Gad, president, and interim chief executive officer, said in a press release. “Y-mAbs is committed to working closely with the FDA on their review of the biologic license application for omburtamab ahead of their decision. We want to thank all of the patients, caregivers, and healthcare providers who participated in the studies of this life-threatening condition.”
The investigational monoclonal antibody targets immune checkpoint B7-H3, which is known to be expressed in a number of cancer types.
Following the biologics license application (BLA) that was submitted to the FDA in August 2020, a refusal to file letter was penned by the FDA in October 2020 with regard to 131I-omburtamab in the aforementioned patient population.2 The regulatory organization indicated that after a preliminary review, sections of the Chemistry, Manufacturing and Control module within the BLA needed further detail, although no requests for additional non-clinical data were requested at the time.
Data from study 03-133 indicated that in a population of 107 patients who were given 2 doses of 131I-omburtamab, the median OS was 50.8 months, although the final median had not been reached at the time of the updated readout.4 This was a favorable outcome when considering the median OS of 47.1 months that was reported during a previous readout in a population of 93 patients.