Autoimmune/Cholestatic/Biliary Diseases News

PHILADELPHIA — Primary sclerosing cholangitis is an independent risk factor for colorectal cancer, with an “almost three-times higher” risk among patients without inflammatory bowel disease vs. the general population, a researcher reported.

The long-stagnant primary biliary cholangitis drug market has seen renewed attention in the past 3 months with the addition of two FDA-approved therapy options as well as a costly ruling from an FDA advisory committee.

An FDA advisory committee voted against approval for obeticholic acid in primary biliary cholangitis without cirrhosis or compensated cirrhosis with portal hypertension, citing “concern for real possible harm”.

Gilead’s Livdelzi, a selective peroxisome proliferator-activated receptor delta agonist, has received accelerated FDA approval for the treatment of primary biliary cholangitis, according to a company release.

The FDA has updated the label indication for Livmarli for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis, expanding the patient population to include infants aged 12 months and older.

Maralixibat improved cholestatic pruritis and reduced serum bile acids in pediatric patients with progressive familial intrahepatic cholestasis, regardless of disease genotype, according to data from the MARCH-PFIC trial.

Interim analyses of two phase 2b studies of volixibat support its use as potential treatment for patients with primary biliary cholangitis and primary sclerosing cholangitis, according to a press release from Mirum Pharmaceuticals.

In a Healio video exclusive, Mitchell L. Shiffman, MD, reports that U.S. patients with severe alcohol-associated hepatitis treated with larsucosterol experienced a “statistically significant” reduction in 90-day mortality — more than 50%.

The FDA granted accelerated approval to Ipsen Pharma’s Iqirvo 80 mg, a first-in-class oral, once-daily peroxisome proliferator-activated receptor agonist for the treatment of primary biliary cholangitis, according to a company release.

Researchers identified bile duct dysplasia or cancer in most explanted livers with an indication of biliary dysplasia, although as many as 41% had no signs of neoplasia, according to a study of patients with primary sclerosing cholangitis.