Hereditary Angioedema | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2015

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.