The US Food and Drug Administration (FDA) has accepted for review clinical stage biopharmaceutical company Intercept Pharmaceuticals' New Drug Application and granted Priority Review for obeticholic acid (OCA) for the treatment of primary biliary cirrhosis (PBC).
OCA is being developed to treat PBC patients with an inadequate therapeutic response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA), the only drug currently approved to treat PBC. The FDA has set a target date of February 29, 2016 to take action under the Prescription Drug User Fee Act (PDUFA).
"Despite current treatment, PBC remains a leading cause of liver transplant among women, so there is a clear ongoing high unmet medical need for new therapies for patients with PBC," said Mark Pruzanski, M.D., president and chief executive officer.
"Priority review designation accelerates the FDA review timelines, potentially bringing Intercept closer to its goal of delivering the first new medicine for PBC in close to 20 years. We look forward to working with FDA to bring OCA to PBC patients in need as soon as possible."
The FDA grants Priority Review to medicines that, if approved, would be a significant improvement in the safety or effectiveness of treatment, prevention or diagnosis of serious diseases. A priority review designation means FDA's goal is to take action on the marketing application within six months of acceptance as compared to 10 months under standard review. OCA was previously granted Fast Track designation by FDA. A drug development program with Fast Track designation is afforded greater access to FDA for the purpose of expediting the drug's development, review and potential approval.
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 approximately one in 1,000 women over the age of 40. PBC is a leading cause of liver transplant among women in the United States. In Europe, the disease accounts for approximately half of liver transplants due to cholestatic diseases and 6 per cent of all liver transplants.