FDA Advisory Committee Votes Unanimously that Y-mAbs Lacks Sufficient Evidence for Approval of Brain Cancer Drug

Rare Daily Staff

A U.S. Food and Drug Advisory Committee voted 16-0 that Y-mAbs Therapeutics failed to provide sufficient evidence that its experimental brain cancer therapy omburtamab improves overall survival.

“Y-mAbs is committed to working for closely with the FDA on their review of the Biologic License Application of omburtamab ahead of their decision,” said Thomas Gad, president, and interim CEO of Y-mAbs.

The Oncologic Drugs Advisory Committee reviewed data from omburtamab’s clinical development program with a focus on study 03-133 (a pivotal phase 1 study) and study 101 (a pivotal phase 2 study) as well as the historical control group.

Neuroblastoma is a rare type of brain cancer that usually begins in very early nerve cells, before birth. This type of cancer starts in the sympathetic nervous system, which is responsible for controlling involuntary bodily functions such as breathing, heart rate, and digestion, and usually leads to tumors in the abdomen, as well as the spine, chest, neck, or pelvis.

Omburtamab is a radiolabeled monoclonal antibody 8H9(131I-8H9) which targets B7-H3–expressing cells in solid tumors, including embryonal tumors, carcinomas, sarcomas, and brain tumors.  In vitro/ex vivo modeling has shown that omburtamab binds to an FG loop-dependent conformation on the B7-H3 molecule, a domain critical for its biologic function.

Omburtamab is a promising investigational agent for radioimmunotherapy of leptomeningeal metastases, Diffuse Intrinsic Pontine Glioma (DIPG), and malignant ascites.

The FDA has accepted Y-mAbs’ application for market approval of omburtamab under Priority Review with an action target date of November 30, 2022. The FDA is not bound by the Advisory Committee’s recommendations but generally takes the recommendation into consideration when making its decision.

Researchers at MSK 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.