Market Spotlight: Hereditary Angioedema
Market Spotlight: Hereditary Angioedema
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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, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
Author: Informa Pharma Intelligence
Publisher: Meddevicetracker
- Description
- Contents
Overview
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.
- Datamonitor Healthcare 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.
- Half of the industry-sponsored drugs in active clinical development for HAE are in Phase II, with only one drug in Phase III. 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 III trial results for Orladeyo, topline Phase Ib/II trial results for STAR-0215, and a data exclusivity expiration for Ruconest.
- The overall likelihood of approval of a Phase I autoimmune/immunology-other asset is 19.5%, and the average probability a drug advances from Phase III is 66.7%. Drugs, on average, take 10.0 years from Phase I to approval, compared to 9.3 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 51% of trials in Phase I–II, and 49% 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 39 trials.
- Takeda leads industry sponsors with the highest overall number of clinical trials for HAE, 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 inhibitors
10 Bradykinin receptor antagonist
11 EPIDEMIOLOGY
15 MARKETED DRUGS
18 PIPELINE DRUGS
22 RECENT EVENTS AND ANALYST OPINION
22 IONIS PKK-LR for HAE (March 29, 2021)
23 Garadacimab for HAE (March 1, 2021)
24 Orladeyo for HAE (March 1, 2021)
25 KVD900 for HAE (February 9, 2021)
27 Garadacimab for HAE (June 8, 2020)
30 KEY UPCOMING EVENTS
31 KEY REGULATORY EVENTS
31 BioCryst & Celgene Suffer English Funding Setback For Rare Disease Drugs
31 New EU Approvals
31 Icatibant Accord Among Eight New Products To Win EMA Nod
31 Rare Disease Approval For Takeda
32 CHMP Recommends BioCryst’s Orladeyo For EU-Wide Approval
32 Orladeyo Receives Approval For HAE In Japan
32 Takeda’s Takhzyro Gets Nod In China
32 BioCryst’s Oral Option To Take On Injectables In HAE Prophylaxis Market
33 UK Early Access For BioCryst’s Hereditary Angioedema Drug
34 PROBABILITY OF SUCCESS
35 LICENSING AND ASSET ACQUISITION DEALS
35 Pharming Signs License Agreement With NewBridge Pharmaceuticals
35 Pharming And Orchard Seek Stem Cell Solution To Hereditary Angioedema
35 Catabasis Acquires Quellis And Its HAE Candidate
36 REVENUE OPPORTUNITY
38 CLINICAL TRIAL LANDSCAPE
39 Sponsors by status
40 Sponsors by phase
41 Recent events
43 BIBLIOGRAPHY
45 APPENDIX
LIST OF FIGURES
14 Figure 1: Trends in prevalent cases of HAE, 2019–28
18 Figure 2: Overview of pipeline drugs for HAE in the US
18 Figure 3: Pipeline drugs for HAE, by company
19 Figure 4: Pipeline drugs for HAE, by drug type
19 Figure 5: Pipeline drugs for HAE, by classification
23 Figure 6: IONIS PKK-LR for HAE (March 29, 2021): Phase II – ISIS 721744-CS2
27 Figure 7: KVD900 for HAE (February 9, 2021): Phase II – On-Demand Treatment
29 Figure 8: Garadacimab for HAE (June 8, 2020): Phase II – CSL312_2001
30 Figure 9: Key upcoming events in HAE
34 Figure 10: Probability of success in the autoimmune/immunology-other pipeline
38 Figure 11: Clinical trials in HAE
38 Figure 12: Top 10 drugs for clinical trials in HAE
39 Figure 13: Top 10 companies for clinical trials in HAE
39 Figure 14: Trial locations in HAE
40 Figure 15: HAE trials status
41 Figure 16: HAE trials sponsors, by phase
LIST OF TABLES
12 Table 1: Prevalent cases of HAE, 2019–28
16 Table 2: Marketed drugs for HAE
20 Table 3: Pipeline drugs for HAE in the US
22 Table 4: IONIS PKK-LR for HAE (March 29, 2021)
23 Table 5: Garadacimab for HAE (March 1, 2021)
24 Table 6: Orladeyo for HAE (March 1, 2021)
25 Table 7: KVD900 for HAE (February 9, 2021)
28 Table 8: Garadacimab for HAE (June 8, 2020)
36 Table 9: Historical global sales, by drug ($m), 2016–20
37 Table 10: Forecasted global sales, by drug ($m), 2021–25
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