DelveInsight’s “Hereditary Angioedema Pipeline Insight, 2025,” analysis delivers comprehensive insights about 20+ organizations and 30+ pipeline drugs in the Hereditary Angioedema pipeline landscape. It encompasses the Hereditary Angioedema pipeline drug profiles, including clinical and nonclinical stage products. It additionally covers the Hereditary Angioedema pipeline therapeutics assessment by product classification, stage, route of administration, and molecule classification. It further highlights the inactive pipeline products in this therapeutic domain.
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Primary Findings from the Hereditary Angioedema Pipeline Analysis
On 19 September 2025, BioCryst Pharmaceuticals conducted a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies. APeX-A, 7353-312, represents a single-arm, open-label, multicenter study. The study will be conducted in countries where berotralstat is not available either commercially or via another mechanism and is planned in countries where pediatric participants have been enrolled in BCX7353-304 (Study 304).
On 17 September 2025, KalVista Pharmaceuticals Ltd. announced a study of KVD900-303 is an open-label, multicenter clinical trial in patients aged 2 to 11 years old with HAE Type I or II.
DelveInsight’s Hereditary Angioedema pipeline analysis demonstrates a robust therapeutic domain with 20+ active organizations working to develop 30+ pipeline therapies for Hereditary Angioedema treatment.
Prominent Hereditary Angioedema Organizations include Corcept Therapeutics, BioCryst Pharmaceuticals, KalVista Pharmaceuticals, Pharvaris, BioMarin Pharmaceutical, Ionis Pharmaceuticals, Inc., and Intellia Therapeutics and additional entities.
Notable Hereditary Angioedema Pipeline Therapeutic Candidates include NTLA-2002, Donidalorsen, STAR-0215, Garadacimab, TAK-743 300 mg, ADX-324, CSL312, TAK-667, Lanadelumab, KVD900 and additional compounds.
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The Hereditary Angioedema Pipeline Analysis delivers a disease overview, pipeline scenario, and therapeutic assessment of the principal pipeline therapies in this therapeutic domain. The Hereditary Angioedema Pipeline Analysis additionally highlights the unmet needs with respect to Hereditary Angioedema.
Understanding Hereditary Angioedema
Hereditary angioedema represents a disorder characterized by recurrent episodes of severe swelling (angioedema). The most common areas of the body to develop swelling are the limbs, face, intestinal tract, and airway. Minor trauma or stress may trigger an attack, but swelling frequently occurs without a known trigger. Episodes involving the intestinal tract cause severe abdominal pain, nausea, and vomiting.
Hereditary Angioedema Emerging Therapeutic Candidates Profile
KVD900: KalVista Pharmaceuticals
KVD900 represents a candidate from our oral portfolio of plasma kallikrein inhibitors. It exhibits high solubility and high permeability and is uniquely suited for on-demand treatment of HAE attacks, with rapid uptake into the plasma and high plasma concentrations. Data in phase II clinical trial for KVD900 is anticipated in the fourth quarter of 2020. Currently, the therapeutic candidate is in the Phase III stage of development for the treatment of Hereditary angioedema.
PHA121: Pharvaris
PHA121 represents a novel small molecule with drug-like properties. In preclinical studies, PHA121 demonstrates highly potent and selective competitive antagonism of the B2 receptor, and shows rapid and potent activity on oral dosing in a bradykinin-mediated disease model. It is currently in phase II stage of development.
BMN 331: BioMarin Pharmaceutical
BMN 331 represents a gene therapy product candidate for HAE. It is currently in Phase I stage of development.
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The Hereditary Angioedema Pipeline Analysis Provides Insights into
The analysis delivers detailed insights about organizations developing therapies for the treatment of Hereditary Angioedema with aggregate therapies developed by each organization.
It assesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Hereditary Angioedema Treatment.
Hereditary Angioedema Organizations are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
Hereditary Angioedema therapeutic candidates under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, different mechanism of action, and molecular classification.
Detailed examination of collaborations (organization-organization collaborations and organization-academia collaborations), licensing agreements and financing details for future advancement of the Hereditary Angioedema marketplace.
Hereditary Angioedema Organizations
Corcept Therapeutics, BioCryst Pharmaceuticals, KalVista Pharmaceuticals, Pharvaris, BioMarin Pharmaceutical, Ionis Pharmaceuticals, Inc., and Intellia Therapeutics and additional entities.
Hereditary angioedema pipeline analysis provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Infusion, Intradermal, Intramuscular, Intranasal, Intravenous, Oral, Parenteral, Subcutaneous, Topical
Hereditary Angioedema Products have been categorized under various Molecule classifications such as
Gene therapies, Small molecule, Vaccines, Polymers, Peptides, Monoclonal antibodies
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Parameters of the Hereditary Angioedema Pipeline Analysis
Coverage- Global
Hereditary Angioedema Organizations- Corcept Therapeutics, BioCryst Pharmaceuticals, KalVista Pharmaceuticals, Pharvaris, BioMarin Pharmaceutical, Ionis Pharmaceuticals, Inc., and Intellia Therapeutics and additional entities.
Hereditary Angioedema Pipeline Therapeutic Candidates- NTLA-2002, Donidalorsen, STAR-0215, Garadacimab, TAK-743 300 mg, ADX-324, CSL312, TAK-667, Lanadelumab, KVD900 and additional compounds.
Hereditary Angioedema Therapeutic Assessment by Product Classification: Single-agent, Combination, Single-agent/Combination
Hereditary Angioedema Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
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Content Organization
- Introduction
- Executive Summary
- Hereditary angioedema: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Hereditary angioedema – DelveInsight’s Analytical Perspective
- In-depth Commercial Assessment
- Hereditary angioedema Collaboration Deals
- Late Stage Products (Phase III and NDA)
- KVD900: KalVista Pharmaceuticals
- Mid Stage Products (Phase II)
- PHA121: Pharvaris
- Early Stage Products (Phase I)
- BMN 331: BioMarin Pharmaceutical
- Pre-clinical and Discovery Stage Products
- Drug Name: Company Name
- Inactive Products
- Hereditary angioedema Key Companies
- Hereditary angioedema Key Products
- Hereditary angioedema- Unmet Needs
- Hereditary angioedema- Market Drivers and Barriers
- Hereditary angioedema- Future Perspectives and Conclusion
- Hereditary angioedema Analyst Views
- Hereditary angioedema Key Companies
- Appendix
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