According to a story from Medscape, US Food and Drug Administration (FDA) has recently issued a new restriction on the use of obeticholic acid (marketed as Ocaliva). The agency now says that the treatment is contraindicated in patients who have primary biliary cholangitis and advanced cirrhosis (liver scarring). This info has been adding to the official prescribing info and to the drug’s boxed warning label.
About Primary Biliary Cholangitis (PBC)
Primary biliary cholangitis, less commonly referred to as primary biliary cirrhosis, is an autoimmune disease that affects the liver. It is most characterized by progressive damage to the bile ducts, which over time allows bile and other toxic substances to build up in the liver. The disease can ultimately progress to liver scarring and cirrhosis. The precise cause of the disease remains unknown; a genetic predisposition, perhaps involving the POGLUT1 gene, is a risk factor, as is the presence of another autoimmune disease. Females are also much more likely to get primary biliary cholangitis. Symptoms include reduced bone density, skin lesions, fatigue (sometimes severe), jaundice, abdominal swelling, hepatic encephalopathy, and enlarged spleen. Treatment for the disease may include the drug Ursodiol, vitamin supplementation, and liver transplant in severe cases. Some patients experience slowed disease progression to the extent that their lifespan and quality of life are not significantly affected. To learn more about primary biliary cholangitis, click here.
Avoiding Safety Risks
The reason that the FDA has issued this restriction on the use of this medicine is due to a total of 25 reports of major liver injury or liver failure in patients that were using it as directed. Obeticholic acid was approved five years ago under Accelerated approval protocols to be used in combination with ursodeoxycholic acid (UDCA) in adult patients that failed to respond to UDCA on its own or could not tolerate UDCA.
To be clear, the agency still believes that use of obeticholic acid is well worth the risk in primary biliary cholangitis patients that do not have cirrhosis. In 18 of the documented cases, cirrhosis and primary biliary cholangitis were present, and ten of them may have had portal hypertension. These patients saw rapid progression of their disease even though their clinical prognosis would not have predicted it.
The FDA reminds physicians that it is paramount to determine if a patient has advanced cirrhosis before beginning a course of treatment with the drug. In addition, patients that use it should be monitored closely and discontinued in any patient whose cirrhosis becomes advanced.
