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Hereditary Angioedema Emerging Drugs and Key Players, 2020

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Hereditary Angioedema Emerging Drugs and Key Players, 2020

August 17
15:32 2020
Hereditary Angioedema Emerging Drugs and Key Players, 2020

DelveInsight Business Research LLP
“Hereditary Angioedema Pipeline Insight, 2020” report by DelveInsight outlays comprehensive insights of present clinical development scenario and growth prospects across the Hereditary Angioedema market. A detailed picture of the Hereditary Angioedema pipeline landscape is provided, which includes the disease overview and Hereditary Angioedema treatment guidelines.

Hereditary Angioedema (HAE) is a rare genetic disorder caused by the deficiency in functional C1 inhibitor (C1INH) that results in recurrent attacks of localized subcutaneous or mucosal edema, most commonly affecting the skin, intestines, upper respiratory tract, and oropharynx.


Hereditary angioedema with normal C1 inhibitor (HAE type III) is clinically characterized by recurrent angioedema affecting the skin, gastrointestinal tract, and larynx. Skin swellings are the most frequent symptoms of HAE type III. Most often they occur on the face, less frequently at the extremities, and only in rare cases at the genitals.

The landscape of therapeutic options for patients with bradykinin-mediated angioedema has changed dramatically in the last decade. Ongoing research promises even greater change in the foreseeable future. Given the economic and psychosocial burdens for patients living with angioedema, effective therapies with novel mechanisms will offer more choices for patients and physicians, and it will also provide greater flexibility in routes of administration. Ultimately, gene therapy strategies may offer a more definitive durable treatment obviating the need for chronic repeated medication use, though the safety and tolerability of such approaches remains largely unknown.


Key players, such as BioCryst Pharmaceuticals, Ionis Pharmaceuticals, Attune Pharma, etc., are involved in developing therapies for the treatment and prevention of HAE. Several potential therapies for HAE are under investigation. These include newer agents that had shown its efficacy in the treatment and prevention of HAE, including BCX7353 (BioCryst Pharmaceuticals), IONIS-PKK-LRx (Ionis Pharmaceuticals), and ATN-249 (Attune Pharma), which are undergoing evaluation in HAE.

Berinert is a selective inhibitor of plasma kallikrein, a precursor of the inflammatory molecule bradykinin. The potential treatment is being developed as a liquid for acute treatment of HAE, and as a capsule to prevent attacks. In December 2019, BioCryst submitted a new drug application to the U.S. Food and Drug Administration seeking approval of oral, once-daily berotralstat for the prevention of HAE attacks. The company plans to submit a marketing authorization application for berotralstat for the prevention of HAE attacks with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and the European Medicines Agency (EMA) in the first quarter of 2020. The company also investigating oral liquid formulation of the molecule in phase II clinical study for acute HAE.

IONIS-PKK-LRx is a generation 2+ ligand-conjugated antisense (LICA) drug designed to reduce the production of prekallikrein, or PKK, to treat patients with hereditary angioedema, or HAE. PKK plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE. Currently, the molecule is in phase II clinical trial.

ATN-249 is an oral, novel small molecule kallikrein inhibitor that was optimized for potency and pharmacokinetics. ATN-249 has been tested in phase I studies, where 249 achieved high-blood levels and was shown to be safe and well-tolerated. The company is also developing a molecule for the HAE, the product name is not reveled by the company yet.


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Table of contents

1. Report Introduction

2. Hereditary Angioedema 

2.1. Overview

2.2. History 

2.3. Hereditary Angioedema Symptoms

2.4. Causes


2.6. Hereditary Angioedema Diagnosis 

2.6.1. Diagnostic Guidelines

3. Hereditary Angioedema Current Treatment Patterns

3.1. Hereditary Angioedema Treatment Guidelines

4. Hereditary Angioedema – DelveInsight’s Analytical Perspective

4.1. In-depth Commercial Assessment

4.1.1. Hereditary Angioedema companies collaborations, Licensing, Acquisition -Deal Value Trends Assessment Summary

4.1.2. Hereditary Angioedema Collaboration Deals Company-Company Collaborations (Licensing / Partnering) Analysis Company-University Collaborations (Licensing / Partnering) Analysis Hereditary Angioedema Acquisition Analysis

5. Therapeutic Assessment

5.1. Clinical Assessment of Pipeline Drugs 

5.1.1. Assessment by Phase of Development

5.1.2. Assessment by Product Type (Mono / Combination) Assessment by Stage and Product Type

5.1.3. Assessment by Route of Administration Assessment by Stage and Route of Administration

5.1.4. Assessment by Molecule Type Assessment by Stage and Molecule Type

5.1.5. Assessment by MOA Assessment by Stage and MOA

5.1.6. Assessment by Target Assessment by Stage and Target

6. Hereditary Angioedema Late Stage Products (Phase-III)

7. Hereditary Angioedema Mid Stage Products (Phase-II)

8. Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Hereditary Angioedema Discontinued Products

13. Hereditary Angioedema Product Profiles

13.1. Drug Name: Company 

13.1.1. Product Description Product Overview Mechanism of action

13.1.2. Research and Development Clinical Studies

13.1.3. Product Development Activities Collaboration Agreements Acquisition Patent Detail

13.1.4. Tabulated Product Summary General Description Table

Detailed information in the report

14. Hereditary Angioedema Key Companies

15. Hereditary Angioedema Key Products

16. Dormant and Discontinued Products

16.1. Dormant Products

16.1.1. Reasons for being dormant

16.2. Discontinued Products 

16.2.1. Reasons for the discontinuation

17. Hereditary Angioedema Unmet Needs

18. Hereditary Angioedema Future Perspectives

19. Hereditary Angioedema Analyst Review  

20. Appendix

21. Report Methodology

21.1. Secondary Research

21.2. Expert Panel Validation 

Media Contact
Company Name: DelveInsight
Contact Person: Priya Maurya
Email: Send Email
Phone: +919650213330
City: Albany
State: New York
Country: United States

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