This Market Spotlight report covers the Hereditary Angioedema market, comprising key pipeline and marketed drugs, clinical trials, upcoming and regulatory events, patent information, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.

Key Takeaways

• Datamonitor Healthcare estimates that in 2017, there were approximately 402,700 prevalent cases of hereditary angioedema worldwide, and forecasts that number to increase to 440,600 prevalent cases by 2026.
• The approved drugs in the hereditary angioedema space target complement proteins, androgen receptors, follicle-stimulating hormone, gonadotropin-releasing hormone, progesterone receptor, bradykinin B2 receptor, and the kinin-kallikrein system. These are commonly administered via the intravenous or subcutaneous routes, with one product being available in an oral formulation.
• There are only seven drugs in active clinical development for hereditary angioedema, with five drugs in Phase I and two in Phase III.
• Therapies in mid-to-late-stage development for hereditary angioedema focus on targets such as the kinin-kallikrein system and complement proteins. These drugs are administered via the subcutaneous and oral routes.
• High-impact upcoming events for drugs in the hereditary angioedema space comprise topline Phase III trial results for BCX7353, topline Phase II trial results for KVD900, and a data exclusivity expiration for Ruconest.
• The overall likelihood of approval of a Phase I autoimmune/immunology-other asset is 20.7%, and the average probability a drug advances from Phase III is 69.4%. Drugs, on average, take 9.7 years from Phase I to approval, compared to 8.7 years in the overall autoimmune/immunology space.
• There have been only three licensing and asset acquisition deals involving hereditary angioedema drugs during 2014–19. The $125m licensing agreement signed in 2016 between Pharming and Bausch Health, for the acquisition of North American commercialization rights for Ruconest, was the largest deal during the period.
• The distribution of clinical trials across Phase I–IV indicates that the majority of trials for hereditary angioedema have been in the late phases of development, with 55% of trials in Phase III–IV, and 45% in Phase I–II.
• The US has a substantial lead in the number of hereditary angioedema clinical trials globally. The UK leads the major EU markets, while Israel has the top spot in Asia.
• Clinical trial activity in the hereditary angioedema space is dominated by completed trials. Takeda has the highest number of completed trials for hereditary angioedema, with 25 trials.
• Takeda leads industry sponsors with the highest number of clinical trials for hereditary angioedema, followed by BioCryst.