On January 29, Intercept Pharmaceuticals, Inc. (Intercept), headquartered in New York City, NY announced that its investigational new drug obeticholic acid (OCA) has received breakthrough therapy designation (BTD) from the U.S. Food and Drug Administration (FDA) for the treatment of patients with nonalcoholic steatohepatitis (NASH) with liver fibrosis.

NASH is a liver disease characterized by the presence of excessive fat, inflammation and damage in the liver.  It is affects 2 to 5 percent of Americans.  NASH is usually asymptomatic at early stage.  Patients begin to have symptoms, such as fatigue, weight loss, and weakness once the disease is more advanced or progressed to cirrhosis.  The underlying cause of NASH is unknown.  However, there is correlation between the development of NASH and metabolic syndromes such as obesity, diabetes and hyperlipidemia.  Currently, there are no specific therapies for NASH, rather than recommendations of losing weight, eating a balanced diet, increasing exercise, avoiding alcohol, etc.  Once progressed to advanced cirrhosis, liver transplantation is the only available treatment option.

OCA is a bile acid analog and a potent first-in-class agonist of the farnesoid X receptor (FXR). FXR is a bile acid receptor highly expressed in the liver and intestines.  The major function of FXR is to suppress bile acid biosynthesis from cholesterol and regulate hepatic triglyceride levels.  According to the company’s website, OCA is being developed for a variety of chronic liver diseases including primary biliary cirrhosis (PBC), nonalcoholic steatohepatitis (NASH) and primary sclerosing cholangitis (PSC).  If successful in clinical trials, OCA will become the first effective therapy to treat NASH and other liver diseases.


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