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Hereditary Angioedema

15
Pipeline Programs
15
Companies
50
Clinical Trials
3 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
1
3
1
6
3
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
643%
RNA Therapeutic
536%
Monoclonal Antibody
321%
+ 51 programs with unclassified modality

On Market (1)

Approved therapies currently available

CSL Behring
ANDEMBRYApproved
garadacimab
CSL Behring
subcutaneous2025

Competitive Landscape

15 companies ranked by most advanced pipeline stage

CSL Behring
CSL BehringIL - Bradley
7 programs
1
Factor XIIa antagonist monoclonal antibodyPHASE_21 trial
C1 Esterase InhibitorPHASE_2_31 trial
C1 Esterase InhibitorPHASE_31 trial
C1-Esterase InhibitorPHASE_31 trial
CSL312PHASE_31 trial
+2 more programs
Active Trials
NCT03712228Completed44Est. Oct 2021
NCT00168103Completed126Est. Dec 2007
NCT00292981Completed57Est. May 2010
+4 more trials
CSL Seqirus
CSL SeqirusUK - Maidenhead
7 programs
1
1
4
1
GaradacimabPhase 4Monoclonal Antibody
C1 Esterase InhibitorPhase 3
C1-Esterase InhibitorPhase 3
CSL312Phase 3
CSL312Phase 3
+2 more programs
Human BioSciences
Human BioSciencesWV - Martinsburg
1 program
1
IcatibantPhase 41 trial
Active Trials
NCT01457430Completed19Est. Apr 2014
ADARx Pharmaceuticals
ADARx PharmaceuticalsCA - San Diego
1 program
1
ADX-324Phase 33 trials
Active Trials
NCT07428499Recruiting90Est. Jun 2030
NCT06960213Recruiting90Est. Dec 2027
NCT05691361Active Not Recruiting53Est. Dec 2027
Rhine Pharma
Rhine PharmaGermany - Heidelberg
1 program
1
C1-Esterase InhibitorPhase 3
Astria Therapeutics
3 programs
1
1
1
STAR-0215Phase 21 trial
STAR-0215Phase 1/21 trial
STAR-0215Phase 11 trial
Active Trials
NCT05477160Completed41Est. Nov 2023
NCT05695248Completed29Est. Mar 2025
NCT06007677Active Not Recruiting56Est. Mar 2031
Halozyme Therapeutics
1 program
1
CINRYZE with rHuPH20Phase 21 trial
Active Trials
NCT01426763Completed12Est. Nov 2011
BioCryst Pharmaceuticals
14 programs
BerotralstatN/ASmall Molecule1 trial
BCX4161PHASE_11 trial
BCX4161PHASE_11 trial
BCX7353PHASE_11 trial
BCX7353PHASE_11 trial
+9 more programs
Active Trials
NCT04428632Approved For Marketing
NCT02218294Completed7Est. Sep 2014
NCT02125162Completed28Est. Jul 2014
+11 more trials
KalVista Pharmaceuticals
10 programs
InapplicableN/A1 trial
SebetralstatN/ASmall Molecule1 trial
SebetralstatN/ASmall Molecule1 trial
KVD824PHASE_11 trial
KVD824 Prototype 1 modified-release tabletPHASE_11 trial
+5 more programs
Active Trials
NCT07009262Completed88Est. Nov 2025
NCT07216378Available
NCT06628713Approved For Marketing
+7 more trials
Takeda
TakedaTOKYO, Japan
6 programs
C1 esterase inhibitor [human]PHASE_13 trials
CINRYZEPHASE_21 trial
CINRYZE with rHuPH20PHASE_2
C1 esterase inhibitor [human]PHASE_31 trial
LanadelumabPHASE_3Monoclonal Antibody1 trial
+1 more programs
Active Trials
NCT00432510Completed27Est. Feb 2007
NCT00438815Completed113Est. Mar 2009
NCT00462709Completed146Est. Mar 2009
+4 more trials
Ionis Pharmaceuticals
5 programs
DonidalorsenN/ARNA Therapeutic1 trial
DonidalorsenPHASE_2RNA Therapeutic1 trial
DonidalorsenPHASE_2RNA Therapeutic1 trial
DonidalorsenPHASE_3RNA Therapeutic1 trial
DonidalorsenPHASE_3RNA Therapeutic1 trial
Active Trials
NCT06415448Available
NCT04307381Completed20Est. Jan 2025
NCT04030598Completed23Est. Mar 2021
+2 more trials
Pharvaris
PharvarisNetherlands - Leiden
4 programs
DeucrictibantPHASE_21 trial
Deucrictibant low dosePHASE_21 trial
deucrictibant selected dosePHASE_2_31 trial
Deucrictibant, PlaceboPHASE_31 trial
Active Trials
NCT04618211Completed74Est. Mar 2023
NCT05047185Completed34Est. Jun 2025
NCT05396105Enrolling By Invitation150Est. Jun 2027
+1 more trials
Intellia Therapeutics
3 programs
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002N/A1 trial
Biological NTLA-2002PHASE_1_21 trial
NTLA-2002PHASE_31 trial
Active Trials
NCT06262399Enrolling By Invitation100Est. Apr 2040
NCT05120830Active Not Recruiting37Est. Jul 2026
NCT06634420Active Not Recruiting60Est. Sep 2027
CENTOGENE
CENTOGENEGermany - Rostock
1 program
Hereditary Angioedema Kininogen AssayN/A1 trial
Active Trials
NCT04091113Terminated59Est. Dec 2021
BioMarin Pharmaceutical
1 program
Dose 1 of BMN 331PHASE_1_21 trial
Active Trials
NCT05121376Active Not Recruiting44Est. Nov 2028

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
CSL BehringGaradacimab
Human BioSciencesIcatibant
TakedaC1 inhibitor
ADARx PharmaceuticalsADX-324
ADARx PharmaceuticalsADX-324
Intellia TherapeuticsNTLA-2002
KalVista PharmaceuticalsKVD900 150 mg
PharvarisDeucrictibant, Placebo
BioCryst PharmaceuticalsBerotralstat
KalVista PharmaceuticalsKVD900 600 mg
KalVista PharmaceuticalsKVD900 600 mg
Ionis PharmaceuticalsDonidalorsen
Ionis PharmaceuticalsDonidalorsen
BioCryst Pharmaceuticalsberotralstat
CSL BehringCSL312

Showing 15 of 50 trials with date data

Clinical Trials (50)

Total enrollment: 3,620 patients across 50 trials

Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab

Start: Mar 2025Est. completion: Jun 202630 patients
Phase 4Recruiting

Efficacy, Safety and Tolerability of Icatibant for the Treatment of HAE

Start: Dec 2011Est. completion: Apr 201419 patients
Phase 4Completed
NCT00914966TakedaC1 inhibitor

A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE

Start: Aug 2009Est. completion: May 201220 patients
Phase 4Completed

Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)

Start: Apr 2026Est. completion: Jun 203090 patients
Phase 3Recruiting

STOP-HAE: A Phase 3 Study of ADX-324 in HAE

Start: Aug 2025Est. completion: Dec 202790 patients
Phase 3Recruiting

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

Start: Jan 2025Est. completion: Sep 202760 patients
Phase 3Active Not Recruiting

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

Start: Jun 2024Est. completion: Jan 202636 patients
Phase 3Completed
NCT06343779PharvarisDeucrictibant, Placebo

Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Start: Feb 2024Est. completion: Oct 2025134 patients
Phase 3Completed

Berotralstat Treatment in Children With Hereditary Angioedema

Start: Oct 2022Est. completion: Feb 202729 patients
Phase 3Active Not Recruiting

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)

Start: Oct 2022Est. completion: Jun 2026145 patients
Phase 3Active Not Recruiting

PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial

Start: Oct 2022Est. completion: Jun 202611 patients
Phase 3Active Not Recruiting

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

Start: Jul 2022Est. completion: Mar 2027154 patients
Phase 3Active Not Recruiting

OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

Start: Dec 2021Est. completion: Nov 202391 patients
Phase 3Completed

Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies

Start: Jul 2021Est. completion: Aug 2031139 patients
Phase 3Enrolling By Invitation

Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks

Start: Apr 2021Est. completion: Nov 2025171 patients
Phase 3Completed

CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks

Start: Jan 2021Est. completion: Jun 202264 patients
Phase 3Completed

A Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Children

Start: Aug 2019Est. completion: Oct 202121 patients
Phase 3Completed

Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE

Start: Feb 2018Est. completion: Apr 2022121 patients
Phase 3Completed

Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema

Start: Dec 2015Est. completion: Feb 20166 patients
Phase 3Terminated
NCT00748202CSL BehringC1-Esterase Inhibitor

Berinert P Study of Subcutaneous Versus Intravenous Administration

Start: Sep 2008Est. completion: Dec 201024 patients
Phase 3Completed
NCT00438815TakedaC1 esterase inhibitor [human]

Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks

Start: Sep 2006Est. completion: Mar 2009113 patients
Phase 3Completed
NCT00462709TakedaC1 esterase inhibitor [human]

Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks

Start: Jun 2006Est. completion: Mar 2009146 patients
Phase 3Completed
NCT00292981CSL BehringC1 Esterase Inhibitor

C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)

Start: Aug 2005Est. completion: May 201057 patients
Phase 3Completed
NCT01005888TakedaC1 esterase inhibitor [human]

C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks

Start: Mar 2005Est. completion: Aug 200726 patients
Phase 3Completed
NCT05396105Pharvarisdeucrictibant selected dose

Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

Start: Dec 2022Est. completion: Jun 2027150 patients
Phase 2/3Enrolling By Invitation

A Long Term Safety Study of BCX7353 in Hereditary Angioedema

Start: Feb 2018Est. completion: Apr 2022387 patients
Phase 2/3Completed

12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks

Start: Dec 2014Est. completion: Jan 2016110 patients
Phase 2/3Completed
NCT00168103CSL BehringC1 Esterase Inhibitor

Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks

Start: Jun 2005Est. completion: Dec 2007126 patients
Phase 2/3Completed

A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema

Start: Sep 2023Est. completion: Mar 203156 patients
Phase 2Active Not Recruiting
NCT05047185PharvarisDeucrictibant low dose

Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II

Start: Apr 2022Est. completion: Jun 202534 patients
Phase 2Completed

Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

Start: Feb 2021Est. completion: Mar 202374 patients
Phase 2Completed

An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

Start: Apr 2020Est. completion: Jan 202520 patients
Phase 2Completed

A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema

Start: Jan 2020Est. completion: Mar 202123 patients
Phase 2Completed

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

Start: Jul 2019Est. completion: Dec 202084 patients
Phase 2Completed
NCT03712228CSL BehringFactor XIIa antagonist monoclonal antibody

A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE)

Start: Oct 2018Est. completion: Oct 202144 patients
Phase 2Completed

Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE

Start: Nov 2013Est. completion: May 201424 patients
Phase 2Completed

A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase

Start: Sep 2011Est. completion: Nov 201112 patients
Phase 2Completed

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration

Start: Jun 2010Est. completion: Dec 201026 patients
Phase 2Completed

A Study of STAR-0215 in Participants With Hereditary Angioedema

Start: Feb 2023Est. completion: Mar 202529 patients
Phase 1/2Completed

Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients

Start: Dec 2022Est. completion: Dec 202753 patients
Phase 1/2Active Not Recruiting

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

Start: Feb 2022Est. completion: Nov 202844 patients
Phase 1/2Active Not Recruiting

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

Start: Dec 2021Est. completion: Jul 202637 patients
Phase 1/2Active Not Recruiting

A Study of STAR-0215 in Healthy Adult Participants

Start: Jul 2022Est. completion: Nov 202341 patients
Phase 1Completed
NCT05118958KalVista PharmaceuticalsKVD824 Prototype 1 modified-release tablet

Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations

Start: May 2020Est. completion: Dec 202037 patients
Phase 1Completed

A Single and Multiple Doses Safety, Tolerability, Pharmacokinetics and Food Effect Study of KVD824 in Healthy Volunteers

Start: Feb 2019Est. completion: Jun 2019108 patients
Phase 1Completed

A Single Dose Safety, Tolerability, Pharmacokinetic and Food Effect Study of KVD900 (Sebetralstat) in Healthy Volunteers

Start: Jan 2018Est. completion: Sep 201884 patients
Phase 1Completed

A Relative Bioavailability Study of Two Formulations of BCX7353

Start: Feb 2017Est. completion: Sep 201724 patients
Phase 1Completed

A Drug-Drug Interaction Study to Evaluate Drug Transporter Interactions

Start: Feb 2017Est. completion: Aug 201754 patients
Phase 1Completed

An Open-label Drug-Drug Interaction Study to Evaluate the Effect of BCX7353 on Cytochrome P450 Enzyme Activity Using Probe Substrates

Start: Mar 2016Est. completion: Jun 201620 patients
Phase 1Completed

First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of BCX7353 in Healthy Western and Japanese Volunteers

Start: May 2015Est. completion: Dec 2015122 patients
Phase 1Completed

Related Jobs

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

6 late-stage (Phase 3) programs, potential near-term approvals
3 actively recruiting trials targeting 3,620 patients
15 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.