FDA Tells Intercept It Won’t Grant Full Approval to Its Rare Liver Disease Drug

Rare Daily Staff

The U.S. Food and Drug Administration notified Intercept Pharmaceuticals that it would not approve its supplemental New Drug Application seeking full approval for Ocaliva, for the treatment of people with the rare and progressive liver disease primary biliary cholangitis.

The decision follows a September meeting of the FDA’s Gastrointestinal Drugs Advisory Committee, which voted 10-1 against granting Ocaliva full approval.

The FDA told Intercept that it is unable to approve its application in its current form. In a letter to the company, the agency said it was continuing to consider safety data from a study submitted by the company, along with other safety information.

Primary biliary cholangitis (PBC) is a chronic autoimmune disease that affects the bile ducts in the liver and is most prevalent in women over the age of 40. PBC causes bile acid to build up in the liver, resulting in inflammation and scarring (fibrosis), which, if left untreated, can lead to cirrhosis, a liver transplant, or death.

Ocaliva is a farnesoid X receptor (FXR) agonist used for the treatment of adult patients with PBC without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension, either in combination with ursodeoxycholic acid (UDCA) with an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. The FDA granted accelerated approval to Intercept for Ocaliva to treat PBC in 2016.

Ocaliva continues to be available for the treatment of appropriate patients living with PBC in the United States under accelerated approval status.

“We believe in the totality of evidence supporting Ocaliva and intend to work closely with the FDA on next steps,” said Vivek Devaraj, U.S. president and chairman of Intercept.

Intercept said Ocaliva’s safety profile is based on extensive data from long-term clinical-trials, published real-world evidence, and external-control studies, as well as eight years of post-marketing patient experience that collectively spans 42,000 patient-years.