Amatuximab is primarily used in cancer therapy, particularly targeting mesothelin-expressing tumors. Mesothelin is overexpressed in several malignancies including malignant pleural mesothelioma, pancreatic ductal adenocarcinoma, and some ovarian cancers. Amatuximab binds to mesothelin on the surface of cancer cells, which can inhibit tumor growth and promote immune-mediated destruction of the cells through mechanisms like antibody-dependent cellular cytotoxicity (ADCC).

It has been investigated in clinical trials as a targeted treatment to improve outcomes in patients with advanced cancers, especially in combination with chemotherapy. By specifically targeting mesothelin, amatuximab helps spare healthy tissues, reducing off-target effects commonly seen with conventional chemotherapy. Additionally, it serves as a reference antibody in research and development of new mesothelin-targeted therapies, including antibody-drug conjugates and CAR-T cell therapies.