Market Spotlight: Hereditary Angioedema (HAE), Pharma Intelligence
- February 2019 •
- 39 pages •
- Report ID: 5780149 •
- Format: PDF
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.