Omburtamab BLA Re-Submitted to FDA for Pediatric Neuroblastoma

Resubmission has been completed for a biologic license application (BLA) for omburtamab in pediatric patients with central nervous system (CNS)/leptomeningeal metastatic from neuroblastoma following a refusal to file letter from the FDA, according to a press release from Y-mAbs Therapeutics.¹

The application is based on results from 2 phase 2 trials, 101 (NCT03275402) and 03-133 (NCT00089245), with results expected to be published later in 2022.^2,3 In October of 2020, the FDA issued a refusal to file letter based on the need for further detail of chemistry, manufacturing, and control, as well as a clinical module.⁴

“I am excited to see the completion of Y-mAbs’ second BLA submission in neuroblastoma. As children treated for high-risk systemic neuroblastoma potentially experience longer systemic remissions, we expect more patients eventually relapsing with brain metastasis and there is currently no effective therapy beyond surgery and radiotherapy available for these patients.” Thomas Gad, founder, chairman and president at Y-mAbs, said in the press release.

The 03-133 study included 107 evaluable patients who received 2 doses of omburtamab. The median overall survival (OS) was 50.8 months, and the final median had not yet been reached at the time of the updated data readout in June 2019. The prior data readout reported a median OS of 47.1 months in 93 patients.

Investigators also treated 68 patients with other CNS cancers such as metastatic lesions. Patients in this group received a total of 201 injections of omburtamab. Rare self-limited grade 1 or 2 adverse effects (AEs) included fever, headache, and vomiting. A total of 3 injections were associated with grade 3 AEs and necessitated treatment discontinuation; these AEs included chemical meningitis and increased communicating hydrocephalus. Myelosuppression also occurred in patients who underwent craniospinal radiation with dose levels exceeding 60 mCi.

In the 101 study, 17 patients were enrolled. Investigators reported a 12-month OS rate of 87% after a median follow-up of 26 weeks vs 30% in a historical control group that was previously disclosed by Y-mAbs.

“We believe omburtamab can potentially address a significant unmet medical need for children with CNS/leptomeningeal metastasis from neuroblastoma, and we look forward to working with the FDA to bring omburtamab to the appropriate patients. This is a key milestone for families and patients facing CNS/leptomeningeal metastasis from neuroblastoma and for Y-mAbs,” Claus Moller, MD, PhD, chief executive officer at Y-mAbs, concluded.

References

1. Y-mAbs announces submission of omburtamab biologics license application to the FDA. News Release. Y-mAbs Therapeutics. April 1, 2022. Accessed April 4, 2022. https://yhoo.it/3NXVXBJ
2. Y-mAbs announces positive omburtamab clinical data. News Release. Y-mAbs Therapeutics. October 28, 2019. Accessed April 4, 2022. https://bit.ly/3xaoSfw
3. Y-mAbs announces update on naxitamb and omburtamab in neuroblastoma. News Release. Y-mAbs Therapeutics. October 16, 2020. Accessed April 4, 2022. https://bit.ly/3DLvimU
4. Y-mAbs provides regulatory update on omburtamab for the treatment of patients with neuroblastoma. News Release. Y-mAbs Therapeutics. October 5, 2020. Accessed April 4, 2022. https://bit.ly/36MHPKQ