This Market Spotlight report covers the Hereditary Angioedema market, comprising key marketed and pipeline drugs, recent events and analyst opinion, clinical trials, upcoming and regulatory events, probability of success, patent information, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
The publisher estimates that in 2019, using the most commonly cited prevalence estimate of 1:50,000, there were approximately 154,300 prevalent cases of hereditary angioedema (HAE) worldwide, and forecasts that number to increase to approximately 168,100 prevalent cases by 2028.
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 are commonly administered via the intravenous, subcutaneous, and oral routes.
Industry-sponsored drugs in active clinical development for HAE are spread evenly across Phase I and Phase II. Therapies in development for HAE focus on targets such as the kinin-kallikrein system, coagulation factor XII, and bradykinin B2 receptor. The majority of pipeline drugs are administered via the oral route, with the remainder being tested as subcutaneous formulations.
High-impact upcoming events for drugs in the HAE space comprise topline Phase II trial results for IONIS PKK-LR, topline Phase III trial results for Orladeyo, topline Phase Ib/II trial results for QLS-215, and a data exclusivity expiration for Ruconest.
The overall likelihood of approval of a Phase I autoimmune/immunology-other asset is 19%, and the average probability a drug advances from Phase III is 66.7%. Drugs, on average, take 9.8 years from Phase I to approval, compared to 9.2 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 early and midphases of development, with 53% of trials in Phase I-II, and 47% in Phase III-IV.
The US has the highest number of HAE clinical trials globally. Germany leads 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 37 trials.
Takeda leads industry sponsors with the highest overall number of clinical trials for HAE, followed by BioCryst.
Key Topics Covered:
Plasma-derived C1-INH (pdC1-INH)
Recombinant C1-INH (rhC1-INH)
Bradykinin receptor antagonist
RECENT EVENTS AND ANALYST OPINION
KVD900 for HAE (February 9, 2021)
Garadacimab for HAE (June 8, 2020)
Orladeyo for HAE (January 12, 2020)
Orladeyo for HAE (November 6, 2019)
KEY UPCOMING EVENTS
KEY REGULATORY EVENTS
CHMP Recommends BioCryst's Orladeyo For EU-Wide Approval
Orladeyo Receives Approval For HAE In Japan
Takeda's Takhzyro Gets Nod In China
BioCryst's Oral Option To Take On Injectables In HAE Prophylaxis Market
UK Early Access For BioCryst's Hereditary Angioedema Drug
Adalvo Files For Generic Version Of Takeda's Firazyr
BioCryst Pharmaceuticals' berotralstat Among 14 New Hopefuls At EMA
PROBABILITY OF SUCCESS
LICENSING AND ASSET ACQUISITION DEALS
Catabasis Acquires Quellis And Its HAE Candidate
CLINICAL TRIAL LANDSCAPE
Sponsors by status
Sponsors by phase
For more information about this report visit https://www.researchandmarkets.com/r/z931wk
View source version on businesswire.com: https://www.businesswire.com/news/home/20220127005891/en/
Laura Wood, Senior Press Manager
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