On September 28, 2012 amatuximab-a drug designed to help treat patients with mesothelioma-received orphan drug status from the FDA. Orphan drug status simply means a potential drug has received a grant from the FDA to help test the drug in treating rare diseases upon the request of a sponsor.
In this case, Morphotek Inc. has taken on the responsibility of sponsoring amatuximab (MORAb-009). Morphotek Inc. has expressed they are very pleased to receive orphan drug status for amatuximab since it has the potential to provide an additional treatment option for patients suffering from mesothelioma.
Mesothelioma is a type of cancer caused by asbestos. Once asbestos fibers are inhaled or swallowed, they can attach to the lining of the lungs and other organ tissues in the body including the mesothelium. Amatuximab is an investigational antibody that targets the cell surface of these cancerous tissues.
Earlier this year, Morphotek Inc. conducted a clinical trial to evaluate amatuximab in patients with mesothelioma. The objective of the study was to dertermine if amatuximab would aid in progression-free survival (PFS) and overall survival (OS) of mesothelioma patients. PFS is the length of time during and after treatment that a patient lives with cancer, but does not get worse. OS is a term used to describe the chances of survival of cancer patients.
The clinical trial enrolled 89 patients with malignant mesothelioma. All 89 patients received Amatuximab in combination with chemotherapy. Patients who achieved a stable response after receiving the combination of drugs then received amatuximab alone. After 6 months, 52% of patients obtained PFS. The average OS rate of patients was 14.5 months. However, only 29 patients were still alive as of April and only 5 of those patients were continuing to receive amatuximab as maintenance therapy.
Researchers concluded that PFS was not significantly different from what was observed with chemotherapy treatment alone. However, the OS rate of patients observed in the study was considered a benefit. With the aid of the FDA grant, the researchers suggested that the effectiveness of amatuximab in mesothelioma patients will be studied more, hopefully leading to successful results.