- Intercept Pharmaceuticals last week won U.S. approval for a drug aimed at treating primary biliary cholangitis (PBC), a rare liver disease which hasn't had a new treatment in nearly 20 years.
- The FDA granted accelerated approval for obeticholic acid based on a surrogate endpoint, meaning Intercept will have to carry out confirmatory trials to verify the drug actually improves survival or reduces the risk of the risk of cirrhosis in PBC patients.
- Announced after markets closed Friday, news of the approval boosted Intercept shares by nearly 5% in Tuesday trading (U.S. markets were closed on Monday for Memorial Day).
Obeticholic acid, which will be marketed in the U.S. as Ocaliva, was approved both in combination with the existing standard treatment ursodeoxycholic acid (UDCA) and as a monotherapy in adults who can't tolerate UDCA.
Its approval gives PBC patients a new treatment option for the first time since UDCA was approved in 1997.
Shares of Intercept closed at $148.36, up 4.65% from Friday's close.
PBC is a slow-moving, chronic liver disease which damages bile ducts in the liver, causing build-up of bile. This eventually leads to cirrhosis, or scarring, of the liver and can necessitate transplantation. Obeticholic acid, also known as OCA, binds to the farnesoid X receptor (FXR) to increase bile flow out of the liver and reduce production of bile generally.
The FDA granted accelerated approval based on the drug's ability to reduce the levels of a biomarker called alkaline phosphatase. Lower amounts of alkaline phosphastase are though to predict actual clinical benefit in PBC patients, although Intercept's studies have yet to definitively prove that.
Some forecasts have estimated sales of OCA will eventually top $2 billion, but that figure is based on the drug gaining approval for treatment of non-alcholic steatohepatitis (NASH), another liver disease affecting a greater number of people. Enrollment in a phase 2 trial studying OCA will be completed by year end 2016, according to the company.
Intercept said the drug would be available to U.S. patients with PBC in seven to 10 days and that it would be distributed through a specialty pharmacy network. A decision in the EU is expected in late 2016.