FDA grants orphan drug designation to ganitumab for Ewing sarcoma

The FDA granted orphan drug designation to ganitumab for the treatment of Ewing sarcoma, according to the drug’s manufacturer.

“We are very pleased to be granted orphan drug designation for the use of ganitumab (AMG 479, NantCell) in the treatment of Ewing sarcoma.” Patrick Soon-Shiong, MD, chairman and CEO of NantCell, said in a company-issued release. “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.”

Ganitumab is a fully human monoclonal antibody directed against insulin growth factor-1R, which plays a role in the development and progression of many cancers, allowing cancerous cells to resist the cytotoxic properties of chemotherapy or radiation. The drug also is designed to inhibit cancer cell proliferation through disruption of the P13K/Akt and MAPK pathways.

A phase 3 trial is currently evaluating the use of ganitumab to treat patients with newly diagnosed Ewing sarcoma.

“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,” Soon-Shiong said.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.