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.

QUESTIONS ANSWERED

  • 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?

CONTENT HIGHLIGHTS

  • 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

  • Disease Landscape & Forecast
    • Commercial Outlook
      • Key Findings
        • Key Findings
      • COVID-19: Areas of Potential Forecast Impact
      • Drivers and Constraints
        • What Factors Are Driving Sales in PBC?
        • What Factors Are Constraining Sales in PBC?
      • Segment-Specific Trends
        • UDCA Nonresponders
      • Alternative Market Scenarios
        • Alternative Market Scenarios
    • Forecast
    • Etiology and Pathophysiology
      • Disease Overview
      • Etiology
        • Etiology
      • Pathophysiology
      • Key Pathways and Drug Targets
    • Epidemiology Overview
      • Key Findings
      • Epidemiology Populations
        • Diagnosed Prevalent Cases
        • Diagnosed Incident Cases
        • Drug-Treated Cases
    • Current Treatment
      • Key Findings
      • Diagnosis
        • Treatment Providers and Referral Patterns
      • Treatment Goals
      • Key Current Therapies
        • Overview
        • UDCA
        • OCA
        • Fibrates
        • Symptomatic Therapies for PBC
      • Medical Practice
        • Overview
        • Treatment Guidelines
        • Region-Specific Treatment Practice
    • Unmet Need Overview
      • Current and Future Attainment of Unmet Needs in PBC
    • Emerging Therapies Overview
      • Key Findings
      • Key Emerging Therapies
        • PPAR-δ Agonists
        • Recombinant FGF19
        • FXR Agonists
        • Ileal bile acid transporter Inhibitors
        • PPAR-α/δ Agonists
        • NOX 1&4 inhibitors
      • Select Phase II Compounds in Development for PBC
      • Patient Registries
        • Patient Organizations
      • Orphan Drug Designation
        • Orphan Drug Provisions: United States
        • Orphan Drug Provisions: Europe
    • Access & Reimbursement Overview
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
    • Methodology
      • Bottom-Up Forecasting Overview
        • Patient Populations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • General Sources of Data
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Generic Erosion
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Experts Interviewed
      • PBC Bibliography

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

Kanika Ujla is an Analyst for the Infectious, Niche, & Rare Diseases (INRD) team at Decision Resources Group. In this role she supports the team by conducting primary and secondary research across a range of antibacterial and antiviral indications as well as numerous niche and rare diseases. She holds experience in R&D, consulting, business research with healthcare and life sciences companies

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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