Hereditary Angioedema Treatment Landscape: FDA Approves DAWNZERA, First RNA-Targeted Therapy | DelveInsight's Perspective on Market Impact, Competitive Landscape and Pipeline Therapies

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

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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.

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

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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.

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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 [https://www.delveinsight.com/report-store/hereditary-angioedema-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=xpr], 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 [https://www.delveinsight.com/sample-request/hereditary-angioedema-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=xpr].

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



This release was published on openPR.