Market Spotlight: Hereditary Angioedema (HAE)
Market Spotlight: Hereditary Angioedema (HAE)
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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.
- Description
- Contents
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 2016, there were approximately 396,400 prevalent cases of hereditary angioedema worldwide, and forecasts that number to increase to 434,200 prevalent cases by 2025.
- 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.
- Half of the industry-sponsored drugs in active clinical development for hereditary angioedema are in Phase I, with only one drug in the NDA/BLA phase.
- 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 II and Phase III trial results for BCX7353, estimated PDUFA dates for lanadelumab’s BLA and Ruconest’s sBLA, an expected CHMP opinion for lanadelumab, and an estimated patent expiration for Ruconest.
- The overall likelihood of approval of a Phase I hereditary angioedema asset is 64.3%, and the average probability a drug advances from Phase III is 85.7%. Drugs, on average, take 9.2 years from Phase I to approval, compared to 8.7 years in the overall autoimmune/immunology space.
- There have been only seven licensing and asset acquisition deals involving hereditary angioedema drugs during 2013–18. The largest deal was the option agreement signed in 2017 between KalVista and Merck Sharp & Dohme for KVD 001 and KVD 900 for $752m.
- 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. Shire has the highest number of completed trials for hereditary angioedema, with 25 trials.
- Shire leads industry sponsors with the highest number of clinical trials for hereditary angioedema, followed by BioCryst.
TABLE OF CONTENTS
6 OVERVIEW
7 KEY TAKEAWAYS
8 DISEASE BACKGROUND
8 Subtypes
9 TREATMENT
9 C1-INH concentrates
9 Plasma-derived C1-INH (pdC1-INH)
9 Recombinant C1-INH (rhC1-INH)
9 Kallikrein inhibitor
9 Bradykinin receptor antagonist
11 EPIDEMIOLOGY
15 MARKETED DRUGS
18 Approvals by country
21 PIPELINE DRUGS
25 RECENT EVENTS AND ANALYST OPINION
25 Cinryze (SQ) for Hereditary Angioedema (September 11, 2017)
26 BCX7353 for Hereditary Angioedema (May 25, 2017)
28 Lanadelumab for Hereditary Angioedema (May 18, 2017)
29 BCX7353 for Hereditary Angioedema (February 27, 2017)
32 KEY UPCOMING EVENTS
33 PROBABILITY OF SUCCESS
34 LICENSING AND ASSET ACQUISITION DEALS
34 Pharming Regains Rights To Ruconest, Seizing More Control Over Its Destiny
35 PARENT PATENTS
36 REVENUE OPPORTUNITY
37 CLINICAL TRIAL LANDSCAPE
38 Sponsors by status
39 Sponsors by phase
39 Shire Sees Another Way To Compete With CSL Behring In HAE With New Version Of Cinryze
40 Shire CEO Says SHP643 Results Vindicate Dyax Buy, M&A Strategy
40 CSL Behring Nears Market With Easier-To-Administer HAE Prophylactic
41 BIBLIOGRAPHY
41 Prescription information
43 APPENDIX
LIST OF FIGURES
14 Figure 1: Trends in prevalent cases of hereditary angioedema, 2016–25
21 Figure 2: Pipeline drugs for hereditary angioedema in the US
21 Figure 3: Pipeline drugs for hereditary angioedema, by company
22 Figure 4: Pipeline drugs for hereditary angioedema, by drug type
22 Figure 5: Pipeline drugs for hereditary angioedema, by classifications
26 Figure 6: Cinryze (SQ) for hereditary angioedema (September 11, 2017): Phase III – SAHARA (adolescents and adults)
28 Figure 7: BCX7353 for Hereditary Angioedema (May 25, 2017): Phase II – APeX-1
29 Figure 8: Lanadelumab for hereditary angioedema (May 18, 2017): Phase III – HELP
31 Figure 9: BCX7353 for hereditary angioedema (February 27, 2017): Phase II – APeX-1
32 Figure 10: Key upcoming events in hereditary angioedema
33 Figure 11: Probability of success in the hereditary angioedema
34 Figure 12: Licensing and asset acquisition activity involving hereditary angioedema drugs
35 Figure 13: Parent patents in hereditary angioedema
37 Figure 14: Clinical trials in hereditary angioedema
37 Figure 15: Top 10 drugs for clinical trials in hereditary angioedema
38 Figure 16: Top 10 companies for clinical trials in hereditary angioedema
38 Figure 17: Trial locations in hereditary angioedema
39 Figure 18: Hereditary angioedema trials sponsors, by status
39 Figure 19: Hereditary angioedema trials sponsors, by phase
LIST OF TABLES
12 Table 1: Prevalent cases of hereditary angioedema, 2016–25
16 Table 2: Marketed drugs for hereditary angioedema
19 Table 3: Approvals by country for hereditary angioedema
23 Table 4: Pipeline drugs for hereditary angioedema in the US
25 Table 5: Cinryze (SQ) for hereditary angioedema (September 11, 2017)
27 Table 6: BCX7353 for hereditary angioedema (May 25, 2017)
28 Table 7: Lanadelumab for hereditary angioedema (May 18, 2017)
29 Table 8: BCX7353 for Hereditary Angioedema (February 27, 2017)
36 Table 9: Historical global sales, by drug ($m), 2013–17
36 Table 10: Forecasted global sales, by drug ($m), 2018–22
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