We’ve talked before about the enormous potential in the health care industry for innovations that are rapidly shaping and changing how diseases are treated in America. But there are companies in the biotech sector that can get overlooked because they focus on what are called “orphan” diseases.
Orphan diseases are those that affect fewer than 200,000 people (such as Addison’s, Crohn’s or Hodgkin disease) or a common disease that has been ignored (such as tuberculosis, cholera and malaria) because it is far more prevalent in developing countries than in the developed world. It’s a shame, but with these diseases affecting smaller numbers of patients, the big pharmaceutical companies don’t spend the time and resources to research and develop therapies for them, and many have no known cure or treatment option. To help counter this, the U.S. government and other countries around the world provide certain subsidies and enticements that increase profitability for the drug companies that do discover and provide therapies for these orphan conditions.
One such company is Intercept Pharmaceuticals (ICPT). ICPT focuses on the development and commercialization of treatments for chronic liver disease. It’s most advanced programs are focused on the development of modified bile acids that can regulate key aspects of liver functionality through receptors. Its leading product candidate, obeticholic acid (OCA), has been developed to treat primary biliary cirrhosis (PBC), which is a rare and chronic autoimmune liver disease that, if inadequately treated, may eventually lead to cirrhosis, liver failure and death.