The U.S. Food and Drug Administration (FDA) has granted orphan-drug status to Ganitumab, a human monoclonal antibody with anti-cancer properties, to treat patients with Ewing sarcoma.
The decision was welcomed by Patrick Soon-Shiong, MD, chairman and CEO of NantCell, which developed the drug.
“While improvements in treatment options for patients with Ewing sarcoma have improved outcomes, there remains a significant unmet medical need for many patients, particularly those with newly diagnosed metastatic Ewing sarcoma, which is first-line therapy,” Soon-Shiong, whose company is based in Culver City, Calif., said in a news release. “With the FDA’s award in place, we believe we are in a solid position to accelerate the clinical development of this program and address this significant unmet medical need, especially in children afflicted with this disease.”
Ganitumab binds to membrane-bound IGF1R, which plays a role in the development and progression of many cancers, preventing binding of the ligand IGF-1 and the subsequent activation of the PI3K/Akt signaling pathway. Inhibition of this survival signaling pathway may result in the inhibition of tumor cell proliferation and the induction of tumor cell death.
According to the company, IGF-1R’s anti-apoptotic properties allow cancerous cells to resist the cytotoxic properties of chemotherapeutic drugs or radiation therapy. Ganitumab inhibits cancer cell proliferation through disruption of the P13K/Akt and MAPK pathways.
The efficacy of Ganitumab is being examined in a randomized Phase 3 clinical trial (NCT02306161) as an add-on therapy to multi-agent chemotherapy in 330 patients with newly diagnosed metastatic Ewing sarcoma. The primary endpoint of the study is the time to adverse analytic event — defined to be a disease-related event, diagnosis of a second malignant neoplasm or death — for up to 10 years.
The clinical trial is being conducted by the National Cancer Institute in partnership with Children’s Oncology Group and NantCell in 295 clinical sites across the United States and Canada.