Mutations in the gene coding for C1-inhibitor (C1-INH) protein result in hereditary angioedema (HAE), whereby insufficient levels of C1-INH or abnormal forms of the protein ultimately result in leakage of fluid from blood vessels into connective tissue, apparent as body swelling. In HAE, various and typically mild triggers such as dental treatment or a cold may bring about painful swelling attacks, but swelling can also occur spontaneously. The face, abdomen, or other body parts may be affected, including swelling of the throat that can lead to a life-threatening constriction of the airways. On-demand therapies for HAE are available from several drug developers, either replacing C1-INH or targeting further downstream factors and have proved to be efficacious for acute swelling attacks. However, HAE patients currently have limited options for routine prophylaxis. A high unmet need remains for efficacious, safe, and easily administered long-term prophylactic therapies.

Questions Answered:

  • The number of diagnosed HAE prevalent cases will increase slightly over the 2014-2024 forecast period. What is the number of diagnosed HAE prevalent cases in the United States and the five major European markets (France, Germany, Italy, Spain, and the United Kingdom)? What will the number of diagnosed HAE prevalent cases be by 2024?
  • The main treatment goals in HAE are to allow patients to achieve normal life expectancy and to restore a normal quality of life. What is the shape of the current treatment landscape and what are common approaches in HAE medical practice?
  • Unmet needs in HAE span a range of challenging issues. What are the key avenues of HAE clinical research and drug development efforts? What unmet needs may be addressed by mid- to late-stage emerging HAE therapies?
  • Several emerging prophylactic therapies with improved delivery profiles will enter the HAE therapeutic market by 2024. What are experts’ opinions on these novel agents? How will the HAE treatment paradigm change with the introduction of new treatment options?

Scope:

Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.

Primary research: Eight country-specific interviews with thought-leading HAE specialists.

Epidemiology: Total diagnosed prevalent cases of HAE, diagnosed prevalent cases of HAE by disease type.

Emerging therapies: Phase III: 2; Phase II/III: 1; coverage of select preclinical and Phase I products.

Table of contents

  • Hereditary Angioedema - Landscape & Forecast - Disease Landscape & Forecast
    • Executive Summary
      • Introduction
        • Key Parameters of the Hereditary Angioedema Landscape, 2014-2024
        • Key Parameters of the Hereditary Angioedema Landscape, 2014-2024
        • Key Parameters of the Hereditary Angioedema Landscape, 2014-2024
        • Actionable Recommendations for Drug Developers
      • Note About Our Epidemiology Estimates
      • Commercial Outlook
        • Market Outlook
          • Key Findings
          • Hereditary Angioedema Therapies Forecast to Launch
        • Drivers and Constraints
          • What Factors Are Driving the Market for Hereditary Angioedema
          • Emerging Therapies Forecast to Launch for Hereditary Angioedema by 2024
          • What Factors Are Constraining the Market for Hereditary Angioedema
      • Etiology and Pathophysiology
        • Symptoms of Hereditary Angioedema
          • Common Symptoms of Hereditary Angioedema
          • Expert Insight on Symptoms of Hereditary Angioedema
        • Triggers of Attacks in Hereditary Angioedema
          • Common Triggers of Hereditary Angioedema
          • Expert Insight on Triggers of Hereditary Angioedema
        • Pathophysiology of Hereditary Angioedema
          • C1 Inhibitor Complement Pathways
          • C1 Inhibitor Contact and Coagulation Pathways
      • Epidemiology Overview
        • Introduction
          • Disease Definition
          • Key Sources for Hereditary Angioedema Epidemiology and Model Parameters
          • Risk/Protective Factors for Hereditary Angioedema
        • Epidemiology Populations
          • Literature Review
          • Studies Included in the Analysis of Hereditary Angioedema
          • Studied Excluded from the Analysis of Hereditary Angioedema
          • Population and Forecast Parameters
          • Diagnosed Hereditary Angioedema
          • Hereditary Angioedema Subtype
          • Number of Diagnosed Prevalent Cases of Hereditary Angioedema, 2014-2024
          • Age Distribution of U.S. Diagnosed Prevalent Hereditary Angioedema Cases. 2014
          • Age Distribution of Diagnosed Prevalent Hereditary Angioedema Cases in the Major European Markets, 2014
          • Diagnosed Prevalent Cases of Hereditary Angioedema, by Disease Type, 2014-2024
          • Note About Our Epidemiology Estimates
      • Current Treatment Overview
        • Diagnosis
          • Overview
          • Expert Insight on Diagnosis of Hereditary Angioedema
          • Diagnostic Algorithm for Hereditary Angioedema
          • Referral Patterns and Treatment Providers
          • Expert Insight on Treatment Providers in Hereditary Angioedema
        • Treatment Goals
          • Expert Insight on Treatment Goals of Hereditary Angioedema
          • Key Clinical Tial End Points
          • Key Clinical Trial End Points for On-demand Therapies
          • Key Clinical Trial End Points for Prophylactic Therapies
        • Key Current Therapies
          • Overview
          • Key On-Demand Therapies for Hereditary Angioedema
          • Expert Insight on Factors Impacting Prescribing of On-Demand Therapies
          • Expert Insight on Key On-Demand Therapies for Hereditary Angioedema
          • Select Clinical Trial Outcomes of Plasma-Derived C1 Inhibitors for Acute Attacks of Hereditary Angioedema
          • Select Clinical Trial Outcomes of Icatibant for Acute Attacks of Hereditary Angioedema
          • Select Clinical Trial Outcomes of Ecallantide for Acute Attacks of Hereditary Angioedema
          • Select Clinical Trial Outcomes of Recombinant Human C1 Inhibitor for Acute Attacks of Hereditary Angioedema
          • Prophylactic Therapies for Hereditary Angioedema
          • Key Prophylactic Therapies
          • Expert Insight on Key Prophylactic Therapies
          • Select Clinical Trial Outcomes of Key Prophylactic Therapies
        • Medical Practice
          • Treatment Algorithm
          • Treatment Algorithm for Hereditary Angioedema
      • Unmet Need Overview
        • Introduction
          • Key Findings
        • Attainment of Unmet Needs
          • Current Attainment of Unmet Needs in Hereditary Angioedema
          • Unmet Needs for Hereditary Angioedema
          • Expert Insight on Unmet Needs for Hereditary Angioedema
      • Emerging Therapies Overview
        • Introduction
          • Key Findings
          • Expert Insight
          • Key Emerging Therapies for Hereditary Angioedema
          • Key Emerging Therapies for Hereditary Angioedema
          • Treatment Algorithm of Hereditary Angioedema, 2024
        • Late-Phase Pipeline Analysis
          • Emerging Therapies Forecast to Launch for Hereditary Angioedema by 2024
        • Orphan Drug Designation
          • Expanded Market Exclusivity
          • Tax Credits
          • Support During the Drug Approval Process
          • Grants
          • Provisions for Revoking Expanded Market Exclusivity for Orphan Drugs
          • Fee Waivers for Orphan-Drug Development Are Tiered to Company Size
        • Patient Registries and Patient Associations
          • Select Patient Associations for Hereditary Angioedema
        • Key Emerging Therapies
          • Overview
          • Ongoing Clinical Trials for CSL830
          • Expert Insight on CSL-830
          • Outlook
          • Patient Share and Patient Numbers for CSL-830 in 2024
          • Overview of Avoralstat
          • Ongoing Clinical Trial for Avoralstat
          • Overview of BCX-7353
          • Expert Insight on Avoralstat and BCX-7353
          • Outlook
          • Patient Share and Patient Numbers for Avoralstat and BCX-7353 in 2024
          • Overview
          • Ongoing Clinical Trials for DX-2930
          • Expert Insight on DX-2930
          • Outlook
          • Patient Share and Patient Numbers for DX-2930 in 2024
        • Early-Phase Pipeline Analysis
          • Select Early-Stage Emerging Therapies
          • Expert Insight on Select Early-Stage Emerging Therapies
      • Access and Reimbursement Overview
        • Region-Specific Reimbursement Practices
        • Appendix
          • List of Abbreviations
          • Experts Interviewed
          • Bibliography

      Author(s): Jing Wu

      Jing is a Principal business insights analyst in the infectious, niche, and rare diseases team at Decision Resources Group, focusing on a diverse set of key niche and rare diseases.

      Jing completed her M.B.A. at Boston University with concentrations in health sector management and finance. She also holds an M.S. in biomedical science from the University of Massachusetts Medical School and a B.S. in biochemistry from Nanjing University in China. She worked as a research assistant at the Molecular Cardiology Research Institute of Tufts Medical Center, where she was involved in several projects to identify genetic regulators contributing to heart diseases.