Ocaliva receives FDA approval for rare, chronic liver disease
This approval specifically applies to adults who have shown an inadequate response to UDCA. The treatment can also be used as a single therapy for adult patients who cannot tolerate traditional UDCA treatments.
Ocaliva is an oral treatment that joins the farnesoid X receptor (FXR) from the cell nucleus within the intestines and liver. FXR is important for regulating bile acid metabolic pathways. It also raises the bile flow levels from the liver, reducing bile acid production to decrease toxic amounts of bile acids in the liver.
PBC causes the liver’s small bile ducts to be damaged, inflamed and destroyed. This chronic disease traps bile within the liver, harming the liver’s cells over a period of time. As the illness progresses, patients experience cirrhosis, or scarring on the liver. Eventually, the cirrhosis will progress to the point of shutting down all liver functions.
“Patients left untreated -- or who have not responded to UDCA -- are at risk for liver failure and death,” Amy Egan, deputy director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research, said. “Today’s approval of Ocaliva provides an important treatment option for patients living with PBC who have not responded to the only other approved therapy, UDCA.”