The FDA has denied full approval to Intercept Pharmaceuticals' supplemental new drug application for Ocaliva (obeticholic acid) in the treatment of primary biliary cholangitis (PBC). The decision follows a complete response letter issued to Intercept, indicating that the application could not be approved in its current form.
The FDA's decision aligns with the recommendation from the Gastrointestinal Drug Advisory Committee, which voted 13 to 1 against full approval in September. The committee raised concerns about whether the benefits of Ocaliva on clinical outcomes in PBC patients without cirrhosis or with compensated cirrhosis and portal hypertension could be verified using available data.
In its letter, the FDA stated it would continue to consider safety data from Intercept’s postmarketing requirement confirmatory trial, 747-302. This ongoing evaluation suggests the agency is open to the possibility of identifying a potential benefit not observed with other available therapies.
"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 at Intercept, in a press release. "We remain committed to patients living with PBC who have limited treatment options."
Ocaliva received accelerated approval in 2016 for PBC patients who have an inadequate response to ursodeoxycholic acid (UDCA), or are unable to tolerate UDCA. PBC is a chronic liver disease affecting the small bile ducts inside the liver. If untreated, PBC can lead to cirrhosis and liver failure. The prevalence of PBC is estimated to be about 40 to 400 cases per million in different populations.
Under its accelerated approval status, Ocaliva will continue to be available as a second-line option for PBC patients who do not respond adequately to first-line UDCA or cannot tolerate it. The continued availability provides an option for a subset of patients with limited alternatives, while further data on the drug's safety and efficacy are under review.
