FDA Rejects’ Y-mAbs’ Rare Pediatric Cancer Drug

The U.S. Food and Drug Administration issued a complete response letter for Y-mAbs Therapeutics’ Biologics License Application for the investigational medicine 131I-omburtamab for the treatment of CNS/leptomeningeal metastasis from neuroblastoma, a rare pediatric cancer.

Photo: Thomas Gad, president, and interim CEO at Y-mAbs

The letter indicates that the FDA completed the review of the application and determined that it is unable to approve the BLA in its current form. This is consistent with the outcome of the Oncologic Drugs Advisory Committee Meeting in October, which recommended rejection of the candidate. The CRL includes a recommendation for meeting with the agency to discuss an adequate and well-controlled trial design to demonstrate substantial evidence of effectiveness and a favorable benefit-risk profile.

Y-mAbs is assessing the implications of the CRL and its plans for the omburtamab program.

“We are disappointed by the CRL but not surprised based on the outcome of the ODAC meeting on October 28,” said Thomas Gad, president, and interim CEO at Y-mAbs, in a statement. “While we evaluate the implications of the CRL for the future of omburtamab, we are excited about refining our focus primarily to drive growth from Danyelza and validate our SADA platform in the clinic, with the goal of bringing innovative solutions to patients and value to our shareholders.”

Researchers at Memorial Sloan Kettering developed omburtamab, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests in the compound.

Author: Rare Daily Staff