The US Food and Drug Administration (FDA) has granted orphan drug designation to Conatus Pharmaceuticals' drug candidate emricasan for the treatment of liver transplant patients with reestablished fibrosis to delay the progression to cirrhosis and end-stage liver disease.
The status will help the company in speeding up the development of drugs and biologics which might provide benefit to patients suffering from rare diseases or conditions.
Emricasan, a first-in-class, orally active caspase protease inhibitor, 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 the FDA's orphan drug status of emricasan for treatment of liver transplant patients with reestablished fibrosis reinforces the critical need for alternatives to the only currently available treatment, which is a second transplant.
"This designation is a significant step forward for Conatus' clinical development program for emricasan," Mento said.
So far, the company has studied emricasan in more than 500 subjects in ten clinical trials.
In a randomized Phase IIb clinical trial, emricasan showed 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 severe alcoholic hepatitis patients.
Conatus' 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.
The company, which is mainly focused on the development and commercialization of new medicines to treat liver disease, expects to conduct further trials in 2014.
