Overview

This Market Spotlight report covers the Hereditary Angioedema (HAE) market, comprising key marketed and pipeline drugs, recent events and analyst opinion, clinical trials, upcoming and regulatory events, probability of success, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.

• Datamonitor Healthcare estimates that in 2021, using the most commonly cited prevalence estimate of 1:50,000, there were approximately 157,500 prevalent cases (ranging from 55,100 to 787,500 cases) of hereditary angioedema (HAE) worldwide, and forecasts that number to increase to approximately 171,000 prevalent cases (ranging from 59,800 to 854,800 cases) by 2030.
• The approved drugs in the HAE space target complement pathway, androgen receptors, follicle-stimulating hormone, gonadotropin-releasing hormone, progesterone receptor, bradykinin B2 receptor, and the kinin-kallikrein system. These drugs are commonly administered via the intravenous, subcutaneous, and oral routes.
• Industry-sponsored drugs in active clinical development for HAE are evenly spread across Phase I to Phase III. Therapies in development for HAE focus on targets such as the kinin-kallikrein system, coagulation factor XII, and bradykinin B2 receptor. These drugs are administered via the oral and subcutaneous routes. • High-impact upcoming events for drugs in the HAE space comprise topline Phase III trial results for donidalorsen and sebetralstat, topline Phase II trial results for PHVS416, topline Phase Ib/II trial results for STAR-0215, and a data exclusivity expiration for Ruconest.
• The overall likelihood of approval of a Phase I autoimmune/immunology-other asset is 17.9%, and the average probability a drug advances from Phase III is 65.1%. Drugs, on average, take 10.2 years from Phase I to approval, compared to 9.6 years in the overall autoimmune/immunology space.
• The distribution of clinical trials across Phase I–IV indicates that the majority of trials for HAE 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 the highest number of HAE clinical trials globally. Germany and the UK lead the major European 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 clinical trials for HAE, with 39 trials.
• Takeda leads industry sponsors with the highest overall number of clinical trials for HAE, followed by BioCryst.