US-based Conatus Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its drug candidate emricasan to treat liver transplant recipients with re-established fibrosis to delay the progression to cirrhosis and end-stage liver disease.
Emricasan is an orally active caspase protease inhibitor that is being developed to reduce the activity of enzymes that mediate inflammation and cell death (or apoptosis) and treat patients with chronic liver disease and significant unmet medical need.
Conatus co-founder, president and chief executive officer Stephen Mento said: “This designation is a significant step forward for Conatus’s clinical development programme for emricasan.”
So far, emricasan has been studied in more than 500 subjects in ten clinical trials.
In a randomised Phase IIb clinical trial, emricasan showed statistically significant, consistent, rapid and sustained reduction in elevated levels of two major biomarkers of inflammation and cell death that are implicated in the severity and progression of liver disease.
The drug is currently in a Phase IIb trial in patients with acute-on-chronic liver failure (ACLF), as well as a Phase II clinical trial in patients with severe alcoholic hepatitis.
The company’s initial development strategy targets patients with ACLF, chronic liver failure (CLF), and patients who have developed liver fibrosis postorthotopic liver transplant due to Hepatitis C virus infection.
Conatus expects to carry out further trials in 2014.