According to a story from Targeted Oncology, the US Food and Drug Administration has given Orphan Drug designation to ARX788, an experimental therapy. The designation was given for the treatment of HER2+ stomach cancer. This therapy is being developed by the company Ambrx. It is currently being evaluated in a phase I clinical trial of patients living with advanced, previously treated stomach cancer.
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 stomach 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. Males are also more likely to get this cancer, which suggests the possibility that estrogen could be protective. Symptoms of stomach cancer 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 reserved for experimental medicines that are being developed for the treatment of illnesses that are considered rare. This is defined as any disease that affects less than 200,000 people in the US. To qualify, the treatment must either fulfill a currently unmet medical need or offer potential advantages in safety or efficacy over currently available therapies. This designation confers multiple benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven-year period of market exclusivity if the drug gets FDA approval.
About ARX788
ARX788 is classified as an antibody-drug conjugate that targets the HER2 receptor. The therapy has shown encouraging activity in the early trial so far. ARX788 has demonstrated preclinical activity in HER2+ breast cancer as well. This includes patients that have developed resistance to the treatment trastuzumab emtansine. The investigational therapy has also earned Fast Track designation for HER2+ breast cancer.
