As the first agent to be approved for primary biliary cholangitis (PBC), ursodeoxycholic acid (UDCA), which launched nearly two decades ago, transformed the treatment of PBC by greatly reducing mortality rates. Intercept’s novel FXR agonist, obeticholic acid (Ocaliva), was approved in the United States and Europe in 2016 and experienced strong initial uptake, primarily among UDCA nonresponders. However, a black box warning was added to Ocaliva in 2018, and a notable subset of PBC patients are still underserved by UDCA. Given the medical advances in the treatment of other liver disorders, there is renewed interest in the development of new PBC agents to address areas of unmet need. Intercept Pharmaceuticals hopes to capitalize on the need for new PBC agents, specifically for patients who do not respond to UDCA, and pipeline agents have the potential to improve on both UDCA and Ocaliva and thus could gain share in what is poised to become a highly competitive market.


  • How effective are the current therapies, UDCA and Intercept Pharmaceuticals’ Ocaliva, in treating PBC?
  • Given the black box warning it received in 2018, how has the market outlook for Ocaliva in PBC patients changed in the United States and the EU5?
  • With the availability of UDCA and Ocaliva, what areas of unmet need remain in the treatment of PBC? What improvements can emerging agents offer in the management of PBC?
  • What clinical endpoints do emerging agents need to meet in order to compete effectively against UDCA and Ocaliva?


  • Geographies: United States and EU5.
  • Primary Research: Six country-specific interviews with thought-leading hepatologists/ gastroenterologistssupported by survey data collected for this study.
  • Epidemiology: Diagnosed prevalence of primary biliary cholangitis by country, drug-treated cases of primary biliary cholangitis by country.
  • Forecast: Drug-level sales and patient share of key primary biliary cholangitis therapies in 2018 and 2028.
  • Emerging Therapies: Phase II: 10 drugs.

Table of contents

  • Primary Biliary Cholangitis - Landscape & Forecast - Disease Landscape & Forecast

Author(s): Kanika Ujla; Abey John, MPH

Abey John is a medical graduate with a Master’s in Public Health and has been associated with DRG since September 2015. He works with a global team of epidemiologists in performing systematic reviews of assigned diseases and prepare forecast models for clients. He also is involved in producing analyses for pharmaceutical drug developers on the descriptive epidemiology of major drug indications in mature and developing markets and have an overall experience of three years working in different healthcare sectors across the country.

Prior to joining DRG, Abey had been working with Jhpiego (an affiliate of Johns Hopkins Medical University) in implementing Family Planning Health Programs in India with collaborating with the Government of India. He also has worked with a grassroots level NGO as a health team manager which worked for the benefit of the rural population living in the foothills of the Himalayas.

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