ODAC Votes Against Omburtamab for Pediatric Metastatic Neuroblastoma

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