ODAC Votes Against Omburtamab for Pediatric Metastatic Neuroblastoma

In a 16 to 0 vote, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted that sufficient evidence has not been provided to conclude that 131I-omburtamab improves overall survival (OS) for pediatric patients with central nervous system (CNS)/leptomeningeal metastases from neuroblastoma.¹

ODAC reviewed data from the evaluation of omburtamab in the phase 2 101 (NCT03275402) and 03-133 (NCT00089245) trials, as well as the historical control group.

“We are disappointed by the outcome of [the ODAC] 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 of Y-mAbs Therapeutics, Inc., stated in a press release. “Y-mAbs is committed to working closely with the FDA on their review of the biologic license application [BLA] for omburtamab ahead of their decision. We want to thank all of the patients, caregivers, and health care providers who participated in the studies of this life-threatening condition.”

In April 2022, a BLA for omburtamab for pediatric metastatic neuroblastoma was resubmitted to the FDA, based on findings from the 101 and 03-133 trials.² It was accepted for priority review by the FDA in May 2022, with a Prescription Drug User Fee Act target date of November 30, 2022.

Prior to the ODAC meeting, the FDA expressed concern regarding the OS benefit of omburtamab.³

Previously reported data from the 03-133 trial showed that 107 patients with CNS/leptomeningeal metastases from neuroblastoma who received up to 2 doses of omburtamab experienced a median OS of 50.8 months, though the final median OS had not yet been reached.⁴

Previously reported interim results from the 101 study showed that among 17 patients enrolled on the trial, the 12-month OS rate was 87%.⁵

After a BLA for omburtamab was originally submitted to the FDA in August 2020, the regulatory issued a Refusal to File letter following a preliminary review of data submitted for the agent in October 2020.^6,7

In June 2017, the FDA granted a breakthrough therapy designation to omburtamab based on data from study 03-133.⁸

References

1. Y-mAbs announces outcome of FDA Advisory Committee Meeting on omburtamab. News release. Y-mAbs Therapeutics, Inc. October 28, 2022. Accessed October 31, 2022. https://bit.ly/3SRZ8fk
2. Y-mAbs announces submission of omburtamab biologics license application to FDA. News release. Y-mAbs Therapeutics, Inc. April 1, 2022. Accessed October 31, 2022. https://bit.ly/3wXDgrI
3. FDA. October 28, 2022 meeting of the Oncologic Drugs Advisory Committee. October 28, 2022. Accessed October 31, 2022. https://bit.ly/3W7gurg
4. Y-mAbs announces positive omburtamab clinical data. News release. Y-mAbs Therapeutics, Inc. October 28, 2019. Accessed October 31, 2022. https://bit.ly/2YKpw1W
5. Y-mAbs announces update on naxitamab and omburtamab in neuroblastoma. News release. October 16, 2020. Accessed October 31, 2022. https://bit.ly/3uJwfbi
6. Y-mAbs announces completion of submission of omburtamab biologics license application to FDA. News release. Y-mAbs Therapeutics, Inc. August 6, 2020. Accessed October 31, 2022. https://bit.ly/3khYdEQ
7. Y-mAbs provides regulatory update on omburtamab for the treatment of patients with neuroblastoma. News release. Y-mAbs Therapeutics, Inc. October 5, 2020. Accessed October 31, 2022. https://bit.ly/30Dgq7B
8. Burtomab receives breakthrough therapy designation for advanced form of pediatric cancer. News release. Business Wire. June 7, 2017. Accessed October 31, 2022. https://bwnews.pr/2NJPf49