Intercept Pharmaceuticals has filed obeticholic acid on both sides of the Atlantic as a treatment for primary biliary cirrhosis, a rare liver disease that primarily results from autoimmune destruction of the bile ducts.
The New York-based biopharma is seeking approval from the US Food and Drug Administration and the European Medicines Agency for use of obeticholic acid (OCA) alongside ursodeoxycholic acid (UDCA) in adults unresponsive or intolerant to the latter drug.
UDCA is currently the only approved therapy for PBC and is the standard of care for all patients. But the majority continue to experience persistent elevations in the serum marker alkaline phosphatase (ALP), which is linked with increased risk of liver failure, need for liver transplant and death, highlighting the clear need for alternative options, the firm said.
"In each of our PBC clinical trials, OCA has demonstrated the ability to rapidly and sustainably lower ALP and improve bilirubin levels, both when added to UDCA and as monotherapy,” noted Intercept chief executive Mark Pruzanski, highlighting its potential.
OCA has received orphan drug designation for PBC in both the US and Europe and fast track designation for PBC in the US.