Rare Disease Report

FDA Warns of Rare Liver Treatment's Incorrect Dosage

SEPTEMBER 21, 2017
Mathew Shanley
In May 2016, the U.S. Food and Drug Administration (FDA) approved Ocaliva (obeticholic acid) as a treatment for patients with primary biliary cholangitis (PBC).

Today, the FDA released a statement, warning that the liver disease medication is being dosed incorrectly in some patients and could lead to serious liver injury or death. Since Ocaliva’s approval 13 months ago, 11 cases of serious liver injury and 19 cases of death have been reported in patients receiving the drug.

PBC is a chronic liver condition that can lead to inflammation and scarring of the small, or intrahepatic, bile ducts. Inflammation and permanent damage can occur as a result, leading to a buildup of bile and an eventual loss of liver function.

The approved indication for Ocaliva is for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxylcholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.

Healthcare professionals are encouraged to determine the patient’s baseline liver function before starting treatment with Ocaliva. Per the official press release, “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 5mg daily dosing used for other PBC patients, and if needed, can be increased up to a maximum approved dose of 10 mg twice weekly.”

It is recommended that dosing frequencies are reduced to once- or twice-weekly for patients who progress to moderate to severe liver impairment. If liver injury is suspected at all, it is recommended that treatment with Ocaliva is discontinued altogether.

While 30 total cases of liver injury and death in patients receiving Ocaliva have been reported to the FDA’s Adverse Event Reporting System (FAERS), it is important to remember that the number only includes reports submitted to the FDA; there may be additional unreported cases.

Patients being treated with Ocaliva who experience common symptoms of liver injury like diarrhea, weight loss or abdominal pain and swelling are encouraged to immediately contact their healthcare professionals with questions or concerns.

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