As the first agent to be approved for primary biliary cholangitis (PBC), ursodeoxycholic acid (UDCA) was launched nearly two decades ago and transformed the treatment of this disease by significantly reducing mortality rates. However, there remains a notable subset of PBC patients who are currently underserved by UDCA. Now, given the medical advances seen for treatment of other liver disorders, there is renewed interest in the development of new PBC agents to address areas of unmet need in this market. Intercept Pharmaceuticals hopes to capitalize on the need for new PBC agents, specifically for patients who do not respond to UDCA. Intercept’s novel FXR agonist obeticholic acid (Ocaliva) was approved in the United States and Europe in 2016 and is expected to gain market share primarily among UDCA nonresponders. However, pipeline agents have the potential to improve upon both UDCA and Ocaliva, and thus could gain share in what will be a highly contested market.
Markets covered: United States, France, Germany, Italy, Spain, and United Kingdom.
Primary research: Six country-specific interviews with PBC experts. Surveys were conducted in the United States and Europe to determine patient share estimates and treatment rates.
Epidemiology: Diagnosed prevalent cases, incident cases; diagnosed drug-treated cases. Emerging therapies: Phase II: 8 drugs.
Market forecast features: Single-point patient-based market forecast extending for 2026.
Key companies: Intercept Pharmaceuticals, Gilead, NGM Biopharmaceuticals, Novartis, Shire, GSK, CymaBay, and Gilead.
Key drugs: Obeticholic acid (Ocaliva), NGM-282, seladelpar, lopixibat, GSK-2330672, GS-9674.
Recent events: The recent launch of Ocaliva, an innovative new FXR-agonist, has dramatic implications for the market and related agents in earlier-stage development. In this report, we analyze physician perception of Ocaliva, its likely positioning and uptake, and its implications on emerging PBC therapeutics.