Market Spotlight: Hereditary Angioedema (HAE)
Market Spotlight: Hereditary Angioedema (HAE)
$1,318.00
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.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
- 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.
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 BCX7353 for Hereditary Angioedema (September 4, 2018)
26 Cinryze (SQ) for Hereditary Angioedema (September 11, 2017)
28 BCX7353 for Hereditary Angioedema (May 25, 2017)
30 Takhzyro for Hereditary Angioedema (May 18, 2017)
31 BCX7353 for Hereditary Angioedema (February 27, 2017)
34 KEY UPCOMING EVENTS
35 KEY REGULATORY EVENTS
35 Takhzyro Among Hopefuls For EU Approval Recommendation
35 Takhzyro Approved For HAE Attacks With Nearly Spotless Label
35 With Takhzyro Approval, Shire Could Reclaim HAE Prophylaxis Market From CSL Behring
36 PROBABILITY OF SUCCESS
37 LICENSING AND ASSET ACQUISITION DEALS
37 BioCryst Changes Mind On Idera Merger
38 PARENT PATENTS
39 REVENUE OPPORTUNITY
40 CLINICAL TRIAL LANDSCAPE
41 Sponsors by status
42 Sponsors by phase
42 Recent events
45 BIBLIOGRAPHY
45 Prescription information
47 APPENDIX
LIST OF FIGURES
14 Figure 1: Trends in prevalent cases of hereditary angioedema, 2017–26
21 Figure 2: Overview of 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 classification
26 Figure 6: BCX7353 for Hereditary Angioedema (September 4, 2018): Phase II – ZENITH-1
28 Figure 7: Cinryze (SQ) for Hereditary Angioedema (September 11, 2017): Phase III – SAHARA (Adolescents and Adults)
30 Figure 8: BCX7353 for Hereditary Angioedema (May 25, 2017): Phase II – APeX-1
31 Figure 9: Takhzyro for Hereditary Angioedema (May 18, 2017): Phase III – HELP
33 Figure 10: BCX7353 for Hereditary Angioedema (February 27, 2017): Phase II – APeX-1
34 Figure 11: Key upcoming events in hereditary angioedema
36 Figure 12: Probability of success in the hereditary angioedema pipeline
37 Figure 13: Licensing and asset acquisition deals in hereditary angioedema, 2013–2018
38 Figure 14: Parent patents in hereditary angioedema
40 Figure 15: Clinical trials in hereditary angioedema
40 Figure 16: Top 10 drugs for clinical trials in hereditary angioedema
41 Figure 17: Top 10 companies for clinical trials in hereditary angioedema
41 Figure 18: Trial locations in hereditary angioedema
42 Figure 19: Hereditary angioedema trials status
42 Figure 20: Hereditary angioedema trials sponsors, by phase
LIST OF TABLES
12 Table 1: Prevalent cases of hereditary angioedema, 2017–26
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: BCX7353 for Hereditary Angioedema (September 4, 2018)
27 Table 6: Cinryze (SQ) for Hereditary Angioedema (September 11, 2017)
29 Table 7: BCX7353 for Hereditary Angioedema (May 25, 2017)
30 Table 8: Takhzyro for Hereditary Angioedema (May 18, 2017)
31 Table 9: BCX7353 for Hereditary Angioedema (February 27, 2017)
39 Table 10: Historical global sales, by drug ($m), 2013–17
39 Table 11: Forecasted global sales, by drug ($m), 2018–22
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