Intercept Pharmaceuticals,
today announced that its new product obeticholic acid (OCA) has received
"breakthrough therapy designation" from the U.S. Food and Drug
Administration (FDA) for the treatment of patients with nonalcoholic
steatohepatitis (NASH) with liver fibrosis.
Obeticholic Acid (OCA) is
currently being developed for the treatment of several chronic liver diseases,
including primary biliary cirrhosis (PBC), NASH and primary sclerosing
cholangitis (PSC).
Breakthrough therapy
designation for OCA was based on clinical efficacy and safety data from two
placebo-controlled, Phase 2 clinical trials of OCA. A Phase 2 trial in 64
patients with nonalcoholic fatty liver disease (NAFLD) assessed the impact of
OCA treatment on insulin sensitivity (published in Gastroenterology in June
2013). The FLINT Phase 2 trial in 283 patients with NASH assessed the impact of
Obeticholic Acid (OCA) treatment on liver histology and fibrosis (published in
The Lancet in November 2014).
"We are very pleased to
have received breakthrough therapy designation for NASH with liver fibrosis, as
it will enable us to work closely with FDA to finalize the design of our Phase
3 program," said Mark Pruzanski, M.D., President and Chief Executive
Officer of Intercept. "This designation underscores a recognition of the
urgent need to bring novel treatments to NASH patients who have developed liver
fibrosis, which is expected to make this serious disease the leading cause for
liver transplant in just the next few years. As a first-in-class FXR agonist,
we believe OCA has the potential to be an important treatment option for
patients with no currently approved medicines."
About Intercept and
Obeticholic Acid
Intercept is a
biopharmaceutical company focused on the development and commercialization of
novel therapeutics to treat orphan and more prevalent liver and intestinal
diseases utilizing its expertise in bile acid chemistry. The company's lead
product candidate, obeticholic acid (OCA), is a bile acid analog and
first-in-class agonist of the farnesoid X receptor (FXR). OCA is being
developed for a variety of chronic liver diseases including primary biliary
cirrhosis (PBC), nonalcoholic steatohepatitis (NASH) and primary sclerosing
cholangitis (PSC). OCA has been granted fast track designation by FDA for the
treatment of patients with PBC who have an inadequate response to or are
intolerant of ursodiol. OCA has received orphan drug designation in both the
United States and Europe for the treatment of PBC and PSC. Intercept owns
worldwide rights to OCA outside of Japan, China and Korea, where it has
out-licensed the product candidate to Sumitomo Dainippon Pharma.
About Nonalcoholic
Steatohepatitis:
NASH is a serious chronic
liver disease caused by excessive fat accumulation in the liver that induces
chronic inflammation which leads to progressive fibrosis (scarring) that can
lead to cirrhosis, eventual liver failure and death. There are currently no
drugs approved for the treatment of NASH. Studies have shown that 21-26% of
NASH patients will develop cirrhosis over 8.2 years of follow-up and that
liver-related mortality due to this disease is ten-fold that of the general
population. According to recent epidemiological studies, it is estimated that
more than 10% of the U.S. adult population has NASH with more than 60% of
patients (potentially more than 14 million in total) believed to have liver
fibrosis or cirrhosis due to progression of the disease. The proportion of
liver transplants attributable to NASH has increased rapidly in past years and
by 2020 the disease is projected to become the leading indication for liver
transplant ahead of chronic hepatitis C and alcoholic liver disease. NASH
patients with fibrosis are at greater risk of progressing to cirrhosis, liver
failure and cancer.
