FDA Rejects Approval for Liver Disease Drug, Market Outlook Grim

The U.S. Food and Drug Administration (FDA) made an important decision on Tuesday, denying full approval of Intercept Pharmaceuticals' liver disease drug. The decision raises questions about the drug's future market prospects. Still, Intercept Pharmaceuticals said that despite the FDA's denial of full approval, their drug Ocaliva will continue to receive accelerated approval in the United States. Under the accelerated approval pathway, the FDA requires additional trials to verify a drug's benefits. If data from these trials do not prove a drug's effectiveness, regulators can require companies to withdraw the drug from the market.

Ocaliva received accelerated FDA approval in 2016 to treat patients with primary cholangitis. Primary cholangitis is a rare disease that causes inflammation of the small bile ducts in the liver and eventually destroys them. Because of the rarity of the disease, recruiting patients for confirmatory trials has become very difficult. According to the American Liver Foundation, fewer than 200,000 cases of primary cholangitis are recorded in the United States each year.

Intercept Pharmaceuticals said it will continue to submit safety data on the drug to health regulators. The FDA's decision is in line with recommendations made by an independent panel of experts in September. The panel said the available data did not definitively prove the drug's effectiveness or dispel doubts about its safety.

"I think the future of this drug is now very much up to the applicants and potential patients who will participate in the new trial," Julia Wattacheril, an assistant professor at Columbia University's Wagelow Snake School of Surgery, said before the FDA decision. She also added that it is unclear whether there is the will to devote more resources to gathering further data on the drug.

Ocaliva and ursodecylcholic acid were among the first treatments. Since then, several other drugs, including Gileads Livdeizi, have been made available to patients. In June, the European drug regulator revoked Ocaliva's conditional authorization, saying its efficacy had not been proven.