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

  • Primary Biliary Cholangitis - Landscape & Forecast - Disease Landscape & Forecast
    • Commercial Outlook
      • Key Findings
        • Regional Sales of Key Therapies to Treat PBC: 2018 and 2028
        • PBC SWOT Analysis
      • 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
          • Alternative Scenarios for the PBC Market Through 2028
      • Forecast
        • Forecast Sales of Key Therapies in PBC
      • Etiology and Pathophysiology
        • Disease Overview
          • Etiology
            • Autoimmune Components of PBC
            • Infectious Disease Components of PBC
            • Cytopathic Disease Components of PBC
          • Pathophysiology
            • Pathogenesis of PBC
          • Key Pathways and Drug Targets
            • Drug Targets in PBC
        • Epidemiology Overview
          • Key Findings
            • Epidemiology Populations
              • Disease Definition
              • Methods
              • Sources Used for Diagnosed Prevalent Cases of PBC
              • Number of Diagnosed Prevalent Cases of PBC in the Major Pharmaceutical Markets: 2018-2028
              • Disease Definition
              • Methods
              • Sources Used for Diagnosed Incident Cases of PBC
              • Number of Diagnosed Incident Cases of PBC in the Major Pharmaceutical Markets: 2018-2028
              • Number of Drug-Treated Prevalent Cases of PBC in the Major Pharmaceutical Markets: 2018-2028 (thousands)
          • Current Treatment
            • Key Findings
              • Diagnosis
                • Diagnostic Criteria
                • Treatment Providers and Referral Patterns
                • Expert Insight: Diagnosis of PBC
              • Treatment Goals
                • Key Endpoints Used in Clinical Trials for PBC
              • Key Current Therapies
                • Overview
                • Mechanism of Action of Key Current Drugs or Drug Classes Used for PBC
                • Current Treatments Used for PBC
                • Market Events Impacting the Use of Key Current Therapies in PBC
                • Clinical Trial Outcomes for UDCA
                • Advantages and Disadvantages of UDCA
                • Key Results from Select Clinical Trials Investigating UDCA for the Treatment of PBC
                • Expert Insight: UDCA
                • Clinical Trial Outcomes for OCA
                • Clinical Analysis of OCA for the Treatment of PBC
                • Advantages and Disadvantages of OCA
                • Expert Insight: OCA
                • Expert Insight: Fibrates
                • Fatigue
                • Pruritus
                • Sicca Syndrome
              • Medical Practice
                • Overview
                • Treatment Guidelines
                • PBC Treatment Guidelines
                • Region-Specific Treatment
                • Generalized Treatment Decision Tree for PBC
            • Unmet Need Overview
              • Current and Future Attainment of Unmet Needs in PBC
              • Top Unmet Needs in PBC—Current and Future Attainment
              • Expert Insight: Unmet Need in PBC
            • Emerging Therapies Overview
              • Key Findings
                • Pipeline Trends for PBC
              • Key Emerging Therapies
                • Key Therapies in Development for PBC
                • Estimated Launch Dates of Key Emerging Therapies for the Treatment of PBC
                • Seladelpar Profile
                • Key Ongoing Clinical Trials of Seladelpar in the Treatment of PBC
                • Analysis of the Clinical Development Program for Seladelpar
                • Expert Insight: Seladelpar
                • Expectations for Launch and Sales Opportunity of Seladelpar in PBC
                • NGM-282 Profile
                • Analysis of the Clinical Development Program for NGM-282
                • Expectations for Launch and Sales Opportunity of NGM-282 in PBC
                • FXR Agonists Profile
                • Key Ongoing Clinical Trials of FXR Agonists in the Treatment of PBC
                • Analysis of the Clinical Development Programs for FXR Agonists
                • Expectations for Launch and Sales Opportunity of FXR Agonists in PBC
                • Linerixibat Profile
                • Key Ongoing Clinical Trials of Linerixibat in the Treatment of PBC
                • Analysis of the Clinical Development Program for Linerixibat
                • Expert Insight: Linerixibat
                • Expectations for Launch and Sales Opportunity of Linerixibat in PBC
                • Elafibranor Profile
                • Analysis of the Clinical Development Program for Elafibranor
                • Expert Insight: Elafibranor
                • Expectations for Launch and Sales Opportunity of Elafibranor in PBC
                • Key Results from Select Clinical Trials Investigating Setanaxib for the Treatment of PBC
                • Analysis of the Clinical Development Program for Setanaxib
                • Expert Insight: Setanaxib
                • Expectations for Launch and Sales Opportunity of Setanaxib in PBC
              • Select Phase II Compounds in Development for PBC
                • Patient Registries
                  • Patient Registries for PBC
                  • Prominent PBC Patient Organizations
                • Orphan Drug Designation
                • Access & Reimbursement Overview
                  • Region-Specific Reimbursement Practices
                    • General Reimbursement Environment: United States
                    • General Reimbursement Environment: EU5
                • Methodology
                  • Bottom-Up Forecasting Overview
                    • Patient Populations
                    • Drug-Specific Assumptions
                  • Bottom-Up Forecast Assumptions
                    • General Sources of Data
                    • Drug-Treatment Rate Assumptions in PBC
                    • Patient Share Assumptions in PBC
                    • General Statements About Pricing
                    • Sources for Drug Prices
                    • 2018 Exchange Rates
                    • Price per Treated Day: 2028
                    • Dosing, Days of Therapy, and Compliance
                    • Generic Erosion
                    • Generic Erosion Assumptions
                    • Out-Year Forecasting
                    • Emerging Therapy Prices
                  • Primary Market Research
                    • Experts Interviewed
                • Appendix
                  • Key Abbreviations Related to PBC
                  • Brands, Marketers, and Generic Availability of Key Therapies for PBC, by Market
                  • Experts Interviewed
                  • PBC Bibliography

              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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