FDA Warns of Deaths Linked to Primary Biliary Cholangitis Drug

Obeticholic acid (Ocaliva) for primary biliary cholangitis (PBC) has been tied to 30 severe liver damage cases — including death — in patients since being approved for the condition by the US Food and Drug Administration (FDA) in May 2016.

According to the FDA, the Intercept Pharmaceuticals drug has been identified in 19 cases of death and 11 cases of serious liver damage, and has been excessively dosed in some adult patients with moderate to severe decreases in liver function.

PBC, a chronic liver disease that causes liver bile duct inflammation and damage before destroying its function, has been gauged to be treated by Ocaliva by a blood test that measures liver problems.

Patients suffering from the treatment are receiving a particularly higher frequency of dosing than recommended, according to the FDA.

“Ocaliva may also be associated with liver injury in some patients with mild disease who are receiving the correct dose,” read a statement from the FDA Thursday. “The recommended dosing and monitoring for patients on Ocaliva are described in the current drug label.”

Ocaliva is currently being tested in the phase 3 REGENERATE trial for treating non-cirrhotic nonalcoholic steatohepatitis (NASH) patients with advanced liver fibrosis.

There may be additional cases of harm or death related to Ocaliva, as the FDA’s Adverse Event Report System (FAERS) only includes reports submitted to the administration.

The FDA advised health care professionals determined patient baseline liver function prior to prescribing the treatment.

“Patients with moderate to severe liver impairment (Child-Pugh B and C) should be started on the approved dosing schedule of 5 mg once weekly, rather than the 5 mg daily dosing used for other PBC patients, and if needed, can be increased up to a maximum approved dose of 10 mg twice weekly,” the statement read.

Patients are advised to contact health care professionals with any questions or concerns regarding Ocaliva, and to report new or worsening severe skin itching.

The FDA is currently working with Intercept Pharmaceuticals to address the safety concerns.