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 (HAE) worldwide, and forecasts that number to increase to 440,600 prevalent cases by 2026.
• The approved drugs in the HAE 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 HAE, with five drugs in Phase I and two in Phase III. Therapies in mid-to-late-stage development for HAE 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 HAE 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 21.4%, and the average probability a drug advances from Phase III is 70.0%. Drugs, on average, take 10.0 years from Phase I to approval, compared to 8.8 years in the overall autoimmune/immunology space.
• There have been only three licensing and asset acquisition deals involving HAE drugs during 2014–19. The $125m licensing agreement signed in 2016 between Pharming Group and Bausch Health for the acquisition of North American commercialization rights for Ruconest was the largest deal.
• The distribution of clinical trials across Phase I–IV indicates that trials for HAE have been evenly split between the early and late phases of development, with 51% of trials in Phase III–IV, and 49% in Phase I–II.
• The US has a substantial lead in the number of HAE clinical trials globally. The UK leads the major EU markets, while Israel has the top spot in Asia.
• Clinical trial activity in the HAE space is dominated by completed trials. Takeda has the highest number of completed trials for HAE, with 22 trials.
• Takeda leads industry sponsors with the highest number of clinical trials for HAE, followed by BioCryst.