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.

Questions Answered:

  • For nearly 20 years, there was only therapy approved for PBC. How effective is the mainstay of therapy, UDCA, in treating PBC patients? Does any unmet need remain for patients who respond to UDCA
  • In 2016, Intercept Pharmaceuticals’ Ocaliva became the first-ever FXR-agonist approved for any indication. How will uptake of Intercept Pharmaceuticals’ Ocaliva impact management of PBC patients in the United States and the EU5
  • There are now two agents approved for PBC, and when they are used in combination, they may radically alter clinical outcomes in patients who previously did not show benefit. With the availability of UDCA and Ocaliva, what areas of unmet need remain for the treatment of PBC? What improvements can emerging agents offer in the management of PBC?
  • PBC is a chronic, progressive disease where the most important clinical end points take many years to reach. What clinical end points do emerging agents need to meet in order to compete effectively against UDCA and Ocaliva?

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.


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