Hereditary Angioedema Treatment Landscape: FDA Approves DAWNZERA, First RNA-Targeted Therapy Delveinsight's Perspective On Market Impact, Competitive Landscape And Pipeline Therapies

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"Hereditary Angioedema Market Insight, Epidemiology and Market Forecast report"DelveInsight Business Research's latest report highlights the transformative impact of the FDA's approval of DAWNZERA (donidalorsen) by Ionis Pharmaceuticals, Inc. (NASDAQ: IONS). This landmark approval marks the first RNA-targeted therapy for hereditary angioedema, offering new hope for over 7K patients suffering from this rare, potentially life-threatening genetic condition characterized by recurrent episodes of severe swelling that can affect various parts of the body.

Key Hereditary Angioedema Market Highlights

• DAWNZERA's FDA approval is expected to be a key hereditary angioedema market driver, given its first-in-class status as an RNA-targeted antisense oligonucleotide therapy.

• Hereditary angioedema affects approximately 7K diagnosed patients in the US , with the condition estimated to occur in 1 in 50,000 people worldwide.

• Hereditary Angioedema Companies: Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), Takeda Pharmaceutical Company Limited, BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX), CSL Behring, Pharming Group N.V., Astria Therapeutics, Intellia Therapeutics, KalVista Pharmaceuticals, and others.

• According to Delveinsight's analysis, the hereditary angioedema market size was approximately USD 3 billion in 2023 and is projected to grow significantly during the forecast period (2024–2034).

Market Impact and Hereditary Angioedema Patient Population

According to DelveInsight's Hereditary Angioedema Market Insight, Epidemiology and Market Forecast report , there is a significant unmet hereditary angioedema medical need with approximately 7K0 diagnosed prevalent cases in the US in 2023. The United States accounts for the largest hereditary angioedema market size at approximately 90%, compared to EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.

Hereditary Angioedema is a rare genetic disorder caused by mutations in the SERPING1 gene, resulting in deficient or dysfunctional C1-esterase inhibitor (C1-INH). This deficiency leads to overproduction of plasma kallikrein, which results in excess bradykinin production, causing episodes of severe swelling in various parts of the body including the limbs, face, gastrointestinal tract, and airway. Hereditary angioedema is frequently perceived as affecting females more than males, with approximately 65% of cases occurring in women.

Furthermore, the report highlights that Type I hereditary angioedema occupies the maximum diagnosed patient pool in the US, with approximately 5.5K diagnosed cases in 2023. The hereditary angioedema treatment market is anticipated to witness substantial positive growth owing to better uptake of existing drugs, expected market launches of new therapies, and raised awareness.

Download the Hereditary Angioedema Market report to understand which other factors are driving the therapeutic market @ Hereditary Angioedema Market Trends .

DAWNZERA Treatment Approach

DAWNZERA represents a first-in-class RNA-targeted antisense oligonucleotide designed to target the underlying hereditary angioedema pathophysiology by reducing prekallikrein production. Unlike current treatment approaches, DAWNZERA addresses the root cause by specifically targeting prekallikrein (PKK), which plays an important role in the activation of inflammatory mediators associated with acute HAE attacks. The therapy is administered subcutaneously every 4 weeks, offering hereditary angioedema patients a convenient treatment option that targets hereditary angioedema prevention rather than merely managing acute episodes.

"The DAWNZERA approval represents a significant milestone for the hereditary angioedema community," said an Ionis representative. "This is the first RNA-targeted prophylactic treatment for hereditary angioedema, offering patients a new therapeutic option designed to prevent attacks by targeting the underlying disease mechanism".

DAWNZERA Clinical Validation and Efficacy

The DAWNZERA FDA approval was based on compelling clinical evidence from the pivotal Phase III OASIS-HAE study, which demonstrated statistically significant efficacy in reducing HAE attack rates. Key clinical outcomes include an 81% reduction in mean monthly attack rate compared to placebo over weeks 1 to 25 (p<0.001) for the every 4 weeks dosing regimen. The every 8 weeks dosing showed a 55% reduction (p=0.004).

The median reduction in attack rate from baseline was 90% in the 4-week group, 83% in the 8-week group, and 16% in the placebo group. Additionally, 88% of patients in the DAWNZERA Q4W group achieved clinically meaningful improvement in quality of life measures versus 45% in the placebo group. The hereditary angioedema therapy was well-tolerated with a favorable safety profile, with the most common adverse events being injection site reactions, upper respiratory tract infection, and urinary tract infection.

Hereditary Angioedema Competitive Landscape and Market Positioning

DAWNZERA enters a competitive treatment landscape that includes several established prophylactic therapies for hereditary angioedema prevention. The current hereditary angioedema competitive landscape includes Takeda's TAKHZYRO (lanadelumab), which generated sales of $1.2 billion in 2024 and reduces hereditary angioedema attacks by approximately 90%. BioCryst's oral therapy ORLADEYO (berotralstat) accounted for sales of $438 million in 2024 and represents the first oral prophylactic treatment. CSL's recently approved ANDEMBRY (garadacimab) offers monthly dosing with factor XIIa inhibition.

Current hereditary angioedema treatments fall into three categories for acute treatment: C1-INH concentrates (plasma-derived BERINERT or recombinant RUCONEST), kallikrein inhibitor (Ecallantide), and bradykinin receptor antagonist (FIRAZYR). For prophylaxis, currently approved therapies include CINRYZE, HAEGARDA, TAKHZYRO, and ORLADEYO. DAWNZERA's first-in-class RNA-targeted mechanism provides unprecedented differentiation in this competitive market.

Explore the Hereditary Angioedema Drug Battle: DAWNZERA vs. other emerging therapies. Discover how these breakthrough Hereditary Angioedema therapies compare in efficacy, safety, cost, and market impact @ Hereditary Angioedema Drugs Market .

Emerging Hereditary Angioedema Pipeline Therapies

Several hereditary angioedema companies are actively developing next-generation therapies, including Astria Therapeutics' navenibart (STAR-0215), which is designed to provide long-acting attack prevention with potential for dosing every 3 to 6 months. Initial proof-of-concept results demonstrated a mean monthly attack rate reduction of 90-96% for up to 6 months of follow-up.

Intellia Therapeutics is advancing NTLA-2002, a CRISPR-based gene-editing therapy that has demonstrated up to an 81% reduction in hereditary angioedema attack rates in phase 2 trials with the potential for a one-time treatment approach. KalVista Pharmaceuticals recently gained FDA approval for sebetralstat (EKTERLY), the first oral on-demand therapy for acute HAE attacks. The broader hereditary angioedema pipeline includes companies developing novel mechanisms, including gene editing, siRNA approaches, and oral bradykinin receptor antagonists.

Discover more Hereditary Angioedema pipeline therapies and the clinical development progress they are making @ Hereditary Angioedema Clinical Pipeline .

Industry Expert Perspective

Clinical experts emphasize the significance of RNA-targeted approaches in hereditary angioedema treatment. "The approval of DAWNZERA represents an important advancement in HAE management," commented leading specialists. "By targeting prekallikrein production through antisense oligonucleotide technology, this therapy offers patients a novel mechanism for attack prevention that complements the existing treatment armamentarium for this challenging condition".

Key opinion leaders from various regions have noted the importance of having multiple prophylactic options available, as evidenced by feedback collected by DelveInsight from over 25 KOLs across major markets including the US, EU4, UK, and Japan.

Learn more about what other Industry experts are saying about DAWNZERA FDA Approval and how it will impact the Hereditary Angioedema treatment market @ Key Opinion Leaders on Hereditary Angioedema Market .

Looking Forward

The DAWNZERA approval represents a significant advancement in hereditary angioedema treatment and demonstrates the potential for RNA-targeted therapeutic approaches in rare genetic diseases. DelveInsight's analysts emphasize that the hereditary angioedema therapeutics market is anticipated to witness substantial positive growth owing to better uptake of existing drugs, expected market launches of emerging therapies, and raised awareness. As the pharmaceutical industry continues to focus on precision medicine and novel therapeutic modalities, DAWNZERA's success as the first RNA-targeted hereditary angioedema therapy may pave the way for similar innovations in other rare genetic conditions with significant unmet medical needs.

The approval adds to a rapidly evolving hereditary angioedema treatment landscape that has seen multiple new therapeutic approvals in recent years, offering patients and healthcare providers an expanding arsenal of tools to manage this challenging rare disease.

Table of Contents

1. Key Insights

2. Executive Summary of Hereditary Angioedema

3. Competitive Intelligence Analysis for Hereditary Angioedema

4. Hereditary Angioedema Market Overview at a Glance

5. Hereditary Angioedema: Disease Background and Overview

6. Hereditary Angioedema Patient Journey

7. Hereditary Angioedema Epidemiology and Patient Population

8. Treatment Algorithm, Current Treatment, and Medical Practices

9. Hereditary Angioedema Unmet Needs

10. Key Endpoints of Hereditary Angioedema Treatment

11. Hereditary Angioedema Marketed Products

12. Hereditary Angioedema Emerging Therapies

13. Hereditary Angioedema: Seven Major Market Analysis

14. Attribute analysis

15. 7MM: Market Outlook

16. Access and Reimbursement Overview of Hereditary Angioedema

17. KOL Views

18. Hereditary Angioedema Market Drivers

19. Hereditary Angioedema Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

23. About DelveInsight

About DelveInsight Business Research

DelveInsight is a leading market research and consulting firm specializing in pharmaceutical and healthcare sectors. We provide comprehensive market intelligence, competitive analysis, and strategic insights to guide decision-making for life science companies globally.

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