According to a story from BioSpace, the biopharmaceutical company MacroGenics, Inc. has just announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company’s investigational product candidate margetuximab. This therapy is being developed as a treatment for stomach cancer and cancer of the gastroesophageal junction, the region where the stomach and the esophagus meet.
About Stomach Cancer
Stomach cancer appears in the lining of the stomach; gastric adenocarcinoma is the most common type. In about 60 percent of stomach cancer cases infection with the bacteria Helicobacter pylori is responsible for causing the disease. There are also several other risk factors for this cancer, such as smoking, infection with the Epstein-Barr virus or HIV virus, family history, diabetes, obesity, and a diet high in pickled vegetables, processed or smoked meat, red meat, and salt. Men are also more likely to get stomach cancer, which suggests the possibility that estrogen could be protective. Symptoms include heartburn, abdominal pain, appetite loss, fatigue, weakness, bloating, vomiting, weight loss, and jaundice. It often does not cause symptoms in the early stages, so many patients are diagnosed with advanced disease. Treatment for this disease includes surgery, chemotherapy, radiation, and targeted therapies. The five year survival rate in the US is only 28 percent. To learn more about stomach cancer, click here.
About Orphan Drug Designation
Orphan Drug designation is typically reserved for experimental drugs that are being developed specifically for a disease or condition that is considered rare. This is defined as any illness that affects less than 200,000 people in the US. To qualify, the drug must display advantages in effectiveness or safety over currently available treatments; alternatively, it must satisfy a currently unmet medical need. The designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a period of market exclusivity lasting seven years if the therapy in question gains FDA approval.
Margetuximab is classified as an Fc-engineered monoclonal antibody that targets HER2. The drug is currently being tested in phase 2/3 trials in combination with checkpoint inhibitors and alongside chemotherapy in patients with HER2 positive stomach cancer and gastroesophageal junction cancer.