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Disease

Angioedema

Late-stage therapeutic developmentEmerging research
MeSHAngioedema
1
Publications
21
Clinical trials
2024
Latest publication
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

CRISPR-Cas9 In Vivo Gene Editing of <i>KLKB1</i> for Hereditary Angioedema.

Research2024-02-01The New England journal of medicine

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: High
Key developments
  • 2 regulatory approvals from EMA on record.
  • 4 clinical trials expected to report results, the earliest in Q2 2027.
Upcoming Milestones
Major developments
Major trial resultsHigh impact
An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema
Results posted2026-06-02
Upcoming trial readoutHigh impact
A Phase 3, Randomized, Double-blind, Placebo-controlled, 3-Part Study to Evaluate the Efficacy and Safety of Orally Administered Deucrictibant Extended-release (XR) Tablet for Prophylaxis and Deucrictibant Immediate-release (IR) Capsule for On-demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency
Results expected Q2 20272026-06-29
Important regulatory approvalHigh impact
Approval — Donidalorsen
Dawnzera is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.2026-01-19
Clinical Milestones9View all 9
Results posted 1
Results expected 4
Recently completed 4
+1 more in the activity timeline below
Regulatory Updates2View
  • 2026-01-19Approval — DonidalorsenDawnzera is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.
  • 2025-09-17Approval — SebetralstatEkterly is indicated for symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.&nbsp;
Activity timeline11

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Donidalorsenapproved

Approval — Dawnzera is indicated for routine prevention of recurrent attacks of hereditary angioedem… (2026)

Garadacimabapproved

Approval — Andembry is indicated for routine prevention of recurrent attacks of hereditary angioedem… (2025)

Sebetralstatapproved

Approval — Ekterly is indicated for symptomatic treatment of acute attacks of hereditary angioedema… (2025)

Icatibant acetateapproved

Approval — Icatibant Accord is indicated for symptomatic treatment of acute attacks of hereditary an… (2021)

Lanadelumabapproved

Accelerated approval — Takhzyro is indicated for routine prevention of recurrent attacks of hereditary angioedem… (2018)

Icatibantapproved

Approval — Firazyr is indicated for symptomatic treatment of acute attacks of hereditary angioedema… (2008)

Clinical trials

14 sponsors · 3 new · 4 completed in the last 12 months (net +3)

The current development programme across all trial phases.

Clinical programme
21
All trials
6
Active
16
Late-stage
6
Completed
Late-stage studies

A Phase 3 Extension Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema

Phase 3 · Recruiting · ADARx Pharmaceuticals, Inc.

Lanadelumab in Long-term Prophylaxis of Acquired Angioedema

Phase 4 · Recruiting · Bernstein Clinical Research Center

A Long-term, Open-label Study to Evaluate the Safety and Efficacy of Orally Administered Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Phase 3 · Recruiting · Pharvaris Netherlands B.V.

A Phase 3, Randomized, Double-blind, Placebo-controlled, 3-Part Study to Evaluate the Efficacy and Safety of Orally Administered Deucrictibant Extended-release (XR) Tablet for Prophylaxis and Deucrictibant Immediate-release (IR) Capsule for On-demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency

Phase 3 · Recruiting · Pharvaris Netherlands B.V.

STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema

Phase 3 · Recruiting · ADARx Pharmaceuticals, Inc.

HAELO: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2002 in Participants With Hereditary Angioedema (HAE)

Phase 3 · Active, not recruiting · Intellia Therapeutics
Recruiting

A Phase 3 Extension Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema

Phase 3 · Recruiting · ADARx Pharmaceuticals, Inc.

Lanadelumab in Long-term Prophylaxis of Acquired Angioedema

Phase 4 · Recruiting · Bernstein Clinical Research Center

A Long-term, Open-label Study to Evaluate the Safety and Efficacy of Orally Administered Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Phase 3 · Recruiting · Pharvaris Netherlands B.V.

A Phase 3, Randomized, Double-blind, Placebo-controlled, 3-Part Study to Evaluate the Efficacy and Safety of Orally Administered Deucrictibant Extended-release (XR) Tablet for Prophylaxis and Deucrictibant Immediate-release (IR) Capsule for On-demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency

Phase 3 · Recruiting · Pharvaris Netherlands B.V.

STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema

Phase 3 · Recruiting · ADARx Pharmaceuticals, Inc.
Recently completed

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema Type I or II

Phase 3 · Completed · KalVista Pharmaceuticals, Ltd.

Open-Label Safety, Pharmacokinetic, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With Hereditary Angioedema Type I or II

Phase 3 · Completed · KalVista Pharmaceuticals, Ltd.

An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema

Phase 3 · Completed · CSL Behring

A Phase IV Clinical Study of Evaluating the Safety and Efficacy of Icatibant Injection (Icanticure) in the Treatment of Patients With Bradykinin Induced Angioedema

Phase 4 · Completed · Nang Kuang Pharmaceutical Co., Ltd.

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Lanadelumab for Prevention Against Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH)

Phase 3 · Completed · Shire

A Phase 3, Multicenter, Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Lanadelumab for Prevention Against Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH)

Phase 3 · Completed · Takeda

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2026emaApproval — Donidalorsen· Dawnzera is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. source ↗
2025emaApproval — Sebetralstat· Ekterly is indicated for symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.&nbsp; source ↗
2025emaApproval — Garadacimab· Andembry is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older. source ↗
2021emaApproval — Icatibant acetate· Icatibant Accord is indicated for symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults, adolescents and children aged 2 years and older, with C1 esterase inhibitor deficiency. source ↗
2018emaAccelerated approval — Lanadelumab· Takhzyro is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 2 years and older. source ↗
2008emaApproval — Icatibant· Firazyr is indicated for symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults (with C1-esterase-inhibitor deficiency). source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Research activity

1 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Most influential

CRISPR-Cas9 In Vivo Gene Editing of <i>KLKB1</i> for Hereditary Angioedema.

The New England journal of medicine · 2024 · 126 cites
Recent publications

CRISPR-Cas9 In Vivo Gene Editing of <i>KLKB1</i> for Hereditary Angioedema.

The New England journal of medicine · 2024 · 126 cites
Major themes3
  • Angioedemas, Hereditary1
  • CRISPR-Cas Systems1
  • Gene Editing1
Leading journals1
  • The New England journal of medicine1
Leading researchers8
  • Abdelhady AM1
  • Boiselle C1
  • Butler JS1
  • Clark ED1
  • Cohn DM1
  • Fijen LM1
  • Golden A1
  • Gurugama P1
Affiliations (unnormalised)1
  • Auckland City Hospital (H.J.L.1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Swelling involving the deep DERMIS, subcutaneous, or submucosal tissues, representing localized EDEMA. Angioedema often occurs in the face, lips, tongue, and larynx.

Identifiers
MeSHAngioedema
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.