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Market Spotlight: Hereditary Angioedema

  • ID: 4533540
  • Report
  • November 2019
  • Region: Global
  • 38 pages
  • Datamonitor Healthcare
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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 author 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.
  • The majority of industry-sponsored drugs in active clinical development for HAE are in Phase I. Therapies in development for HAE focus on targets such as the kinin-kallikrein system and bradykinin B2 receptor. The majority of pipeline drugs are administered via the oral route, with two products also being tested in a subcutaneous formulation.
  • High-impact upcoming events for drugs in the HAE space comprise topline Phase II trial results for KVD900, topline Phase III trial results for BCX7353, and a data exclusivity expiration for Ruconest.
  • The overall likelihood of approval of a Phase I autoimmune/immunology-other asset is 22.5%, and the average probability a drug advances from Phase III is 71.1%. Drugs, on average, take 10.0 years from Phase I to approval, compared to 9.0 years in the overall autoimmune/immunology space.
  • There have been only four 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 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. 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 clinical trials for HAE, with 19 trials.
  • Takeda leads industry sponsors with the highest overall number of clinical trials for HAE, followed by BioCryst.
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Key Takeaways

Disease Background
  • Subtypes
  • C1-INH concentrates
  • Plasma-derived C1-INH (pdC1-INH)
  • Recombinant C1-INH (rhC1-INH)
  • Kallikrein inhibitor
  • Bradykinin receptor antagonist

Marketed Drugs

Pipeline Drugs

Recent Events and Analyst Opinion
  • BCX7353 for HAE (May 21, 2019)
  • BCX7353 for HAE (September 4, 2018)
Key Upcoming Events

Key Regulatory Events
  • England's NICE OKs Shire’s Takhzyro For Rare Hereditary Disease
  • Anticancers, Orphans & The First CAR-Ts: New EU Drug Approvals In 2018
Probability of Success

Licensing and Asset Acquisition Deals

Parent Patents

Clinical Trial Landscape
  • Sponsors by status
  • Sponsors by phase
  • Recent events
  • Prescription information

List of Figures
Figure 1: Trends in prevalent cases of HAE, 2017-26
Figure 2: Overview of pipeline drugs for HAE in the US
Figure 3: Pipeline drugs for HAE, by company
Figure 4: Pipeline drugs for HAE, by drug type
Figure 5: Pipeline drugs for HAE, by classification
Figure 6: BCX7353 for HAE (May 21, 2019): Phase III - APeX-2
Figure 7: BCX7353 for HAE (September 4, 2018): Phase II - ZENITH-1
Figure 8: Key upcoming events in HAE
Figure 9: Probability of success in the HAE pipeline
Figure 10: Licensing and asset acquisition deals in HAE, 2014-19
Figure 11: Parent patents in HAE
Figure 12: Clinical trials in HAE
Figure 13: Top 10 drugs for clinical trials in HAE
Figure 14: Top 10 companies for clinical trials in HAE
Figure 15: Trial locations in HAE
Figure 16: HAE trials status
Figure 17: HAE trials sponsors, by phase

List of Tables
Table 1: Prevalent cases of HAE, 2017-26
Table 2: Marketed drugs for HAE
Table 3: Pipeline drugs for HAE in the US
Table 4: BCX7353 for HAE (May 21, 2019)
Table 5: BCX7353 for HAE (September 4, 2018)
Note: Product cover images may vary from those shown
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