US panel supports accelerated approval for Intercept’s trial drug for rare liver condition

pharmafile | April 8, 2016 | News story | Medical Communications, Research and Development, Sales and Marketing Intercept Pharmaceuticals, PDUFA, US FDA, liver disorder, rare disease 

Intercept Pharmaceuticals (Nasdaq: ICPT) said a US Food and Drug Administration (FDA) advisory committee has recommended accelerated approval for its drug to treat a rare liver disease. 

If approved, the drug candidate, Ocaliva (obeticholic acid) would be the first new treatment for primary biliary cholangitis (PBC) in almost two decades, the trial-stage company said in a statement. The committee voted 17 to 0 in favour of Ocaliva on Thursday. 

The vote follows an FDA staff review released earlier this week that had identified no major concerns regarding farnesoid X receptor agonist for use in PBC.  

The target date for the FDA to take action under the Prescription Drug User Fee Act (PDUFA) is May 29, 2016. The FDA is not bound by the Advisory Committee’s recommendations however it takes its advice into consideration when reviewing trial-stage medicines. 

Mark Pruzanski, Chief Executive of Intercept says: “We’re pleased that the Advisory Committee strongly supported the approval of Ocaliva for people living with PBC. Today’s positive recommendation is an encouraging step for the PBC community.” 

Trading in shares of Intercept was halted Thursday ahead of the advisory committee decision. 

PBC is a rare liver disease that primarily results from autoimmune destruction of the bile ducts that transport bile acids out of the liver, resulting in cholestasis. It is primarily a disease of women, afflicting about one in 1,000 women over the age of 40. Since 1988, PBC has been the second-leading overall cause of liver transplant in women in the US, behind hepatitis C. In Europe, the disease accounts for about half of liver transplants due to cholestatic diseases and 6% of all liver transplants. 

Anjali Shukla

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