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Hereditary Angioedema Pipeline: Over 20 Leading Companies Driving Innovation with Next-Gen Therapies | DelveInsight
Hereditary Angioedema Pipeline
DelveInsight's "Hereditary Angioedema Pipeline Insight, 2025" comprehensively analyzes the current clinical landscape and growth prospects in the Hereditary Angioedema market. The report covers disease insights, treatment guidelines, and a detailed pipeline assessment from preclinical to marketed stages. It includes drug mechanisms, clinical studies, regulatory progress, and key developments such as collaborations, mergers, funding, and designations.
For emerging Hereditary Angioedema drugs, the Hereditary Angioedema pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.
Key Takeaways from the Hereditary Angioedema Pipeline Report
• DelveInsight's Hereditary Angioedema Pipeline analysis depicts a robust space with 20+ active players working to develop 30+ pipeline drugs for Hereditary Angioedema treatment.
• The leading Hereditary Angioedema companies include BioCryst Pharmaceuticals, KalVista Pharmaceuticals, Pharvaris, BioMarin Pharmaceutical, Ionis Pharmaceuticals, Inc., Intellia Therapeutics, and others are evaluating their lead assets to improve the Hereditary Angioedema treatment landscape.
• Key Hereditary Angioedema pipeline therapies in various stages of development include BCX7353, KVD900, PHA121, BMN 331, IONIS-PKK-LRx, NTLA-2002, and others.
• In January 2025, Intellia Therapeutics, Inc. announced dosing of the first patient in its global Phase III trial of NTLA-2002 for Hereditary Angioedema (HAE). NTLA-2002 is an investigational in vivo, single-dose CRISPR therapy designed to treat this life-threatening condition. The company expects to complete enrollment by H2 2025 and aims to file a Biologics License Application (BLA) in 2026, targeting a potential U.S. launch in 2027.
• In November 2024, Ionis Pharmaceuticals, Inc. announced that the U.S. FDA has accepted the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted treatment for preventing Hereditary Angioedema attacks in adults and pediatric patients aged 12 and older.
• In September 2024, Astria Therapeutics received Orphan Drug Designation from the FDA for navenibart (STAR-0215) to treat hereditary angioedema (HAE).
• In September 2024, KalVista Pharmaceuticals announced that the FDA accepted the NDA for sebetralstat, an oral plasma kallikrein inhibitor for on-demand treatment of HAE attacks in patients aged 12 and older.
• In September 2024, Intellia Therapeutics announced that Phase II data for NTLA-2002, an investigational CRISPR-based gene editing therapy for HAE, will be presented at the ACAAI Annual Meeting in October 2024.
Request a sample and discover the recent breakthroughs happening in the Hereditary Angioedema pipeline landscape @ https://www.delveinsight.com/report-store/hereditary-angioedema-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr
Hereditary Angioedema Overview
Hereditary angioedema (HAE) is a rare genetic disorder marked by recurrent episodes of severe swelling, or angioedema, most commonly affecting the limbs, face, gastrointestinal tract, and airways. While minor injuries or stress can sometimes trigger an episode, swelling often occurs without an identifiable cause. Intestinal attacks can result in intense abdominal pain, nausea, and vomiting, while airway swelling can be life-threatening due to potential breathing obstruction.
Around one-third of patients may also develop a non-itchy rash (erythema marginatum) during episodes. Symptoms usually start in childhood and tend to worsen around puberty. Without treatment, individuals typically experience attacks every 1-2 weeks, with each episode lasting 3 to 4 days. The frequency and severity of these attacks can vary significantly-even among members of the same family.
Find out more about Hereditary Angioedema medication @ https://www.delveinsight.com/report-store/hereditary-angioedema-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr
Hereditary Angioedema Treatment Analysis: Drug Profile
KVD900 - KalVista Pharmaceuticals
KVD900 is an oral plasma kallikrein inhibitor developed by KalVista Pharmaceuticals, designed specifically for the on-demand treatment of hereditary angioedema (HAE) attacks. It boasts high solubility, excellent permeability, and rapid plasma uptake, allowing for quick and effective symptom control. KVD900 is currently in Phase III clinical trials for HAE.
PHA121 - Pharvaris
PHA121 is an innovative small-molecule B2 receptor antagonist being developed by Pharvaris. It has shown strong potency and selectivity in preclinical studies, effectively targeting bradykinin pathways involved in HAE. The drug is currently advancing through Phase II clinical trials for hereditary angioedema.
Key Hereditary Angioedema Therapies and Companies
• KVD900: KalVista Pharmaceuticals
• PHA121: Pharvaris
• BMN 331: BioMarin Pharmaceutical
• Donidalorsen (IONIS-PKK-LRx): Ionis Pharmaceuticals
• Navenibart (STAR-0215): Astria Therapeutics
Learn more about the novel and emerging Hereditary Angioedema pipeline therapies @ https://www.delveinsight.com/report-store/hereditary-angioedema-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr
Hereditary Angioedema Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.
By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
By Route of Administration
• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravenous
• Oral
• Parenteral
• Subcutaneous
• Topical
By Molecule Type
• Gene therapies
• Small molecule
• Vaccines
• Polymers
• Peptides
• Monoclonal antibodies
Scope of the Hereditary Angioedema Pipeline Report
• Coverage: Global
• Key Hereditary Angioedema Companies: BioCryst Pharmaceuticals, KalVista Pharmaceuticals, Pharvaris, BioMarin Pharmaceutical, Ionis Pharmaceuticals, Inc., Intellia Therapeutics, and others.
• Key Hereditary Angioedema Pipeline Therapies: BCX7353, KVD900, PHA121, BMN 331, IONIS-PKK-LRx, NTLA-2002, and others.
Dive deep into rich insights for drugs used for Hereditary Angioedema treatment; visit @ https://www.delveinsight.com/report-store/hereditary-angioedema-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr
Table of Contents
1. Introduction
2. Executive Summary
3. Hereditary Angioedema Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Hereditary Angioedema Pipeline Therapeutics
6. Hereditary Angioedema Pipeline: Late-Stage Products (Phase III)
7. Hereditary Angioedema Pipeline: Late-Stage Products (Phase III)
8. Hereditary Angioedema Pipeline: Mid-Stage Products (Phase II)
9. Hereditary Angioedema Pipeline: Early Stage Products (Phase I)
10. Therapeutic Assessment
11. Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Key Companies
14. Key Products
15. Unmet Needs
16. Market Drivers and Barriers
17. Future Perspectives and Conclusion
18. Analyst Views
19. Appendix
Contact Us:
Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.
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