dm+d

Unassigned

New Medicines

Primary biliary cholangitis (PBC) in adults with inadequate response to ursodeoxycholic acid (UDCA)

Information

New molecular entity
Genfit
Genfit

Development and Regulatory status

Phase II Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Dual peroxisome proliferator-activated alpha and delta receptor agonist (PPARα/δ agonist)
Autoimmune disease of biliary system; prevalence estimated as 12.9 per 100,000 population in the UK with up to 90% of cases occurring in women [2].
Primary biliary cholangitis (PBC) in adults with inadequate response to ursodeoxycholic acid (UDCA)
Oral

Non-alcoholic steatohepatitis (NASH) - with stage 1, 2 or 3 liver fibrosis

Information

New molecular entity
Genfit
Genfit

Development and Regulatory status

Discontinued
Discontinued
Discontinued
Jul 20Development for NASH discontinued, but will continue for PBC [5].

Category

Dual peroxisome proliferator-activated alpha and delta receptor agonist (PPARα/δ agonist)
Fatty liver disease is divided into alcohol-related fatty liver disease, and non-alcoholic fatty liver disease (NAFLD). The only difference between the two is the alcohol. A threshold of <20 g of alcohol per day in women and <30 g in men is usually used to allow a diagnosis of NAFLD. When inflammation is present, this becomes non-alcoholic steatohepatitis (NASH), which can progress to cirrhosis and hepatocellular carcinoma. In Europe prevalence of NAFLD is 20-30% and NASH is 5% [1].
Non-alcoholic steatohepatitis (NASH) - with stage 1, 2 or 3 liver fibrosis
Oral