+353-1-416-8900REST OF WORLD
+44-20-3973-8888REST OF WORLD
1-917-300-0470EAST COAST U.S
1-800-526-8630U.S. (TOLL FREE)

PRINTER FRIENDLY

Market Spotlight: Hereditary Angioedema

  • ID: 4533540
  • Report
  • March 2021
  • Region: Global
  • 43 pages
  • Datamonitor Healthcare
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.

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.
Note: Product cover images may vary from those shown
OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND
  • Subtypes
TREATMENT
  • C1-INH concentrates
  • Plasma-derived C1-INH (pdC1-INH)
  • Recombinant C1-INH (rhC1-INH)
  • Kallikrein inhibitors
  • Bradykinin receptor antagonist
EPIDEMIOLOGY

MARKETED DRUGS

PIPELINE DRUGS

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
REVENUE OPPORTUNITY

CLINICAL TRIAL LANDSCAPE
  • Sponsors by status
  • Sponsors by phase
  • Recent events
BIBLIOGRAPHY
  • Prescription information
APPENDIX

LIST OF FIGURES
Figure 1: Trends in prevalent cases of HAE, 2019–28
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: KVD900 for HAE (February 9, 2021): Phase II - On-Demand Treatment
Figure 7: Garadacimab for HAE (June 8, 2020): Phase II - CSL312_2001
Figure 8: Orladeyo for HAE (January 12, 2020): Phase III - APeX-J (Japan)
Figure 9: Orladeyo for HAE (November 6, 2019): Phase II/III - APeX-S, Phase III - APeX-2
Figure 10: Key upcoming events in HAE
Figure 11: Probability of success in the autoimmune/immunology-other pipeline
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, 2019–28
Table 2: Marketed drugs for HAE
Table 3: Pipeline drugs for HAE in the US
Table 4: KVD900 for HAE (February 9, 2021)
Table 5: Garadacimab for HAE (June 8, 2020)
Table 6: Orladeyo for HAE (January 12, 2020)
Table 7: Orladeyo for HAE (November 6, 2019)
Table 8: Historical global sales, by drug ($m), 2015–19
Table 9: Forecasted global sales, by drug ($m), 2021–25
Note: Product cover images may vary from those shown
Adroll
adroll