Morphotek, Inc. was awarded Orphan Drug Status for amatuximab earlier this month, sending a ray of hope to early stage mesothelioma patients who are banking on immunotherapy in their battle to survive.
Amatuximab (MORAb-009) is an investigational cancer drug that is made in the laboratory and administered through intravenous drip that goes directly into the blood stream. It has been proven both safe and effective in Phase I and Phase II Clinical Trials.
Orphan Drug Status Speeds Up Development
The Orphan Drug designation by the Federal Drug Administration (FDA) will allow Morphotek to rapidly accelerate the pace of bringing amatuximab to mesothelioma patients looking for new treatment options.
Under the Orphan Drug Act, Morphotek now will qualify for certain financial incentives, including prescription drug-fee waivers, tax credits, and a seven-year marketing exclusivity.
The Orphan Drug Act was designed to encourage pharmaceutical companies to work on therapies for rare diseases like mesothelioma, which is diagnosed in an estimated 3,000 Americans each year. Mesothelioma is caused by exposure to asbestos. A drug can qualify for “orphan” status if it used to treat fewer than 200,000 patients.
The mesothelioma community is especially receptive to orphan drugs because there currently is no cure for this cancer and few viable treatment options. The standard treatment of surgery, radiation therapy and chemotherapy has not changed much in recent years. Even with the best care, a patient with mesothelioma usually doesn’t survive more than two years after the diagnosis.
“We believe that addressing unmet medical needs, no matter the size of the patient population, is essential to the achievement of our human health care mission, which is to give first thought to patients and their families,” said Julia Maltzman, M.D., senior director of clinical development at Morphotek, in a press release. “Ultimately, this antibody has the potential to provide an additional treatment option for patients suffering from an extremely serious disease.”
Amatuximab is an antibody that targets mesothelin, a glycoprotein that becomes over-expressed in several cancers, including pancreatic, ovarian and certain lung cancers. Mesothelin is involved in cell adhesion that encourages new tumor growth. Amatuximab has shown an ability to bind to the mesothelin on cancer cells and trigger an immune response, helping a body’s natural defenses fight off the cancer growth.
Immunotherapy, which has shown great potential in many clinical trials, has been viewed as the key to future mesothelioma treatment. In one clinical trial at the NCI, the drug stabilized the cancer and, at least temporarily, prevented it from spreading in 11 of the 13 mesothelioma patients who received it.
According to Morphotek, both the National Cancer Institute (NCI) and Johns Hopkins University have validated immune-based therapies using mesothelin as a target.
The side effects also have been minimal. Some patients in the trials have experienced flu-like symptoms when the drug is first given, but they did not last very long.
Morphotek, a subsidiary of Eisai, is a pharmaceutical company specializing in the development of antibody and protein product through gene technology. Eisai Inc. began marketing its first product in the United States in 1997.