FDA grants accelerated approval for new liver disease therapy

Adult patients living with primary biliary cholangitis (PBC) — a chronic liver condition marked by the inflammation of the organ's small bile ducts that leads to those ducts becoming damaged and finally destroyed and can lead to liver scarring (cirrhosis ) — will soon have access to a new treatment option.

The U.S. Food and Drug Administration (FDA) has granted accelerated approval for the drug Ocaliva (obeticholic acid), which can be used alone or in combination with ursodeoxycholic acid (UCDA).

Accelerated FDA approval is used for drugs being developed to treat life-threatening conditions.
Because of its chronic nature, PBC typically leads to complete loss of liver function. Ocaliva is an oral therapy that works by increasing bile flow from the liver and supressing bile acid production in the liver.

"Patients left untreated, or who have not responded to UDCA, are at risk for liver failure and death,” Dr. Amy Egan, deputy director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research, said. “Ocaliva provides an important treatment option for patients living with PBC who have not responded to the only other approved therapy, UDCA."

Possible clinical benefits of Ocaliva treatment include transplant-free survival. The drug was the subject of of 216-participant, 12-month clinical trial. Side effects observed included skin itching, fatigue, abdominal pain, joint pain, throat pain, dizziness and constipation; the drug is not recommended for use by patients with complete biliary obstruction.

UDCA, which was greenlit as a PBC treatment by the FDA in 1997, has been found to be effective in 50 percent of patients. In addition to accelerated approval, the FDA also gave Ocaliva orphan drug designation, which is given to drugs being developed for the treatment of rare diseases.
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