Hereditary Angioedema
Pipeline by Development Stage
Drug Modality Breakdown
On Market (2)
Approved therapies currently available
Competitive Landscape
9 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Showing 15 of 46 trials with date data
Clinical Trials (50)
Total enrollment: 2,799 patients across 50 trials
Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab
A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II
Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
Berotralstat Treatment in Children With Hereditary Angioedema
An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)
PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks
CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks
A Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Children
Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE
Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema
Berinert P Study of Subcutaneous Versus Intravenous Administration
Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks
Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks
C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)
C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
A Long Term Safety Study of BCX7353 in Hereditary Angioedema
12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks
Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II
Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II
A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE)
Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE
A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations
A Single and Multiple Doses Safety, Tolerability, Pharmacokinetics and Food Effect Study of KVD824 in Healthy Volunteers
A Single Dose Safety, Tolerability, Pharmacokinetic and Food Effect Study of KVD900 (Sebetralstat) in Healthy Volunteers
A Relative Bioavailability Study of Two Formulations of BCX7353
A Drug-Drug Interaction Study to Evaluate Drug Transporter Interactions
An Open-label Drug-Drug Interaction Study to Evaluate the Effect of BCX7353 on Cytochrome P450 Enzyme Activity Using Probe Substrates
First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of BCX7353 in Healthy Western and Japanese Volunteers
Study to Determine How BCX4161 is Metabolized and Eliminated by the Body
A Study of the Relative Bioavailability of a New Formulation of BCX4161 and the Effect of Food on BCX4161
Pharmacokinetics of C1 Esterase Inhibitor in Hereditary Angioedema Subjects
Oral Berotralstat Expanded Access Program
Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema
Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat
Treatment of Angioedema Attacks in Adolescent and Adult Patients 12 Years and Older With HAE Type I or II With Sebetralstat