Angioedema
Recent clinical, regulatory, research and industry developments relating to this disease.
CRISPR-Cas9 In Vivo Gene Editing of <i>KLKB1</i> for Hereditary Angioedema.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 4 clinical trials expected to report results, the earliest in Q2 2027.
- Q2 2027A Phase 3, Randomized, Double-blind, Placebo-controlled, 3-Part Study to Evaluate the Efficacy and Safety of Orally Administered Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency
- Q4 2027STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema
- Q4 2028A 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
- Q2 2030A Phase 3 Extension Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema
Clinical MilestonesView all 9Hide
- 2026-09-21A Phase 3, Randomized, Double-blind, Placebo-controlled, 3-Part Study to Evaluate the Efficacy and Safety of Orally Administered Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor DeficiencyResults expected Q2 2027
- 2026-09-10STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary AngioedemaResults expected Q4 2027
- 2026-09-04A Phase 3 Extension Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary AngioedemaResults expected Q2 2030
- 2026-05-07A 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 AngioedemaResults expected Q4 2028
- 2026-06-29A Phase 4 Open-label Study to Evaluate the Safety After Switching to CSL312 (Garadacimab) From Current Prophylactic HAE Treatment in Subjects With HAE ≥ 12 Years of AgeCompleted
- 2026-05-27An 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 IICompleted
- 2026-02-10HAELO: 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)Primary completion
- 2026-01-15Open-Label Safety, Pharmacokinetic, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With Hereditary Angioedema Type I or IICompleted
Regulatory UpdatesViewHide
- 2026-01-19Original application approval — DonidalorsenDawnzera is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.
- 2026-09-21ClinicalA Phase 3, Randomized, Double-blind, Placebo-controlled, 3-Part Study to Evaluate the Efficacy and Safety of Orally Administered Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor DeficiencyResults expected Q2 2027
- 2026-09-10ClinicalSTOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary AngioedemaResults expected Q4 2027
- 2026-09-04ClinicalA Phase 3 Extension Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary AngioedemaResults expected Q2 2030
- 2026-06-29ClinicalA Phase 4 Open-label Study to Evaluate the Safety After Switching to CSL312 (Garadacimab) From Current Prophylactic HAE Treatment in Subjects With HAE ≥ 12 Years of AgeCompleted
- 2026-06-02ClinicalAn Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary AngioedemaResults posted
- 2026-05-27ClinicalAn 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 IICompleted
- 2026-05-07ClinicalA 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 AngioedemaResults expected Q4 2028
- 2026-02-10ClinicalHAELO: 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)Primary completion
- 2026-01-19RegulatoryOriginal application approval — DonidalorsenDawnzera is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.
- 2026-01-15ClinicalOpen-Label Safety, Pharmacokinetic, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With Hereditary Angioedema Type I or IICompleted
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Original application approval — Dawnzera is indicated for routine prevention of recurrent attacks of hereditary angioedem… (2026)
Original application approval — Andembry is indicated for routine prevention of recurrent attacks of hereditary angioedem… (2025)
Original application approval — Ekterly is indicated for symptomatic treatment of acute attacks of hereditary angioedema… (2025)
Generic approval — Icatibant Accord is indicated for symptomatic treatment of acute attacks of hereditary an… (2021)
Original application approval — Takhzyro is indicated for routine prevention of recurrent attacks of hereditary angioedem… (2018)
Original application approval — Firazyr is indicated for symptomatic treatment of acute attacks of hereditary angioedema… (2008)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
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
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
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
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.
Reference
Authoritative identity, definition & identifiers.
Swelling involving the deep DERMIS, subcutaneous, or submucosal tissues, representing localized EDEMA. Angioedema often occurs in the face, lips, tongue, and larynx.
- 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.