Hereditary Angioedema (HAE)

21
Pipeline Programs
5
Companies
30
Clinical Trials
1 recruiting
3
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
2
0
5
1
10
3
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Peptide
480%
RNA Therapeutic
120%
+ 16 programs with unclassified modality

On Market (3)

Approved therapies currently available

Ionis Pharmaceuticals
DAWNZERA (AUTOINJECTOR)Approved
donidalorsen
Ionis Pharmaceuticals
subcutaneous2025
Takeda
FIRAZYRApproved
icatibant acetate
Takeda
subcutaneous2011
U
KALBITORApproved
ecallantide
Unknown Company
Plasma Kallikrein Inhibitor [EPC]injection2009

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Takeda
TakedaTOKYO, Japan
14 programs
2
3
1
6
1
CINRYZE 500Phase 31 trial
CINRYZE 500 UPhase 31 trial
DX-2930 - 300mg/2wkPhase 31 trial
ecallantidePhase 3Peptide1 trial
ecallantidePhase 3Peptide1 trial
+9 more programs
Active Trials
NCT04057131Completed179Est. Jul 2024
NCT07293364Not Yet Recruiting514Est. Jan 2027
NCT02741596Completed212Est. Oct 2019
+16 more trials
Ionis Pharmaceuticals
1 program
1
1
DonidalorsenPhase 3RNA Therapeutic1 trial
Active Trials
NCT07298447Not Yet Recruiting20Est. Jun 2029
CSL Behring
CSL BehringIL - Bradley
2 programs
1
CSL312Phase 31 trial
CSL312N/A1 trial
Active Trials
NCT07159464Available
NCT05819775Completed22Est. Nov 2025
Pharvaris
PharvarisNetherlands - Leiden
2 programs
2
DeucrictibantPhase 31 trial
DeucrictibantPhase 31 trial
Active Trials
NCT06679881Recruiting170Est. Dec 2028
NCT06669754Active Not Recruiting81Est. Aug 2026
BioCryst Pharmaceuticals
2 programs
2
BCX7353Phase 21 trial
BCX7353Phase 21 trial
Active Trials
NCT03240133Completed58Est. Jan 2019
NCT02870972Completed75Est. Aug 2017

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
Ionis PharmaceuticalsDonidalorsen
PharvarisDeucrictibant
PharvarisDeucrictibant
CSL BehringCSL312
TakedaIcatibant
TakedaCINRYZE 500 U
TakedaDX-2930
TakedaDX-2930 - 300mg/2wk
TakedaIcatibant
TakedaCINRYZE 500
TakedaIcatibant
TakedaIcatibant
TakedaIcatibant
Takedaecallantide
Takedaecallantide

Showing 15 of 29 trials with date data

Clinical Trials (30)

Total enrollment: 2,485 patients across 30 trials

Donidalorsen Treatment in Children With Hereditary Angioedema

Start: Jan 2026Est. completion: Jun 202920 patients
Phase 3Not Yet Recruiting

Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

Start: Feb 2025Est. completion: Dec 2028170 patients
Phase 3Recruiting

Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

Start: Dec 2024Est. completion: Aug 202681 patients
Phase 3Active Not Recruiting

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

Start: May 2023Est. completion: Nov 202522 patients
Phase 3Completed

A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema

Start: Jan 2021Est. completion: Jul 20212 patients
Phase 3Completed
NCT02865720TakedaCINRYZE 500 U

Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)

Start: Sep 2016Est. completion: Jun 20178 patients
Phase 3Completed

Long-term Safety and Efficacy Study of DX-2930 (SHP643) to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

Start: May 2016Est. completion: Oct 2019212 patients
Phase 3Completed
NCT02586805TakedaDX-2930 - 300mg/2wk

Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

Start: Mar 2016Est. completion: Apr 2017125 patients
Phase 3Completed

A Study of Icatibant for Acute Attacks of Hereditary Angioedema in Japanese Participants

Start: Mar 2015Est. completion: Feb 20168 patients
Phase 3Completed

Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema

Start: Mar 2014Est. completion: May 201712 patients
Phase 3Completed

A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema

Start: Jan 2012Est. completion: Mar 201832 patients
Phase 3Completed

EASSI - Evaluation of the Safety of Self-Administration With Icatibant

Start: Sep 2009Est. completion: Jun 2011151 patients
Phase 3Completed

A Study of Icatibant in Patients With Acute Attacks of Hereditary Angioedema (FAST-3)

Start: Jul 2009Est. completion: Oct 201098 patients
Phase 3Completed

Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)

Start: Apr 2007Est. completion: Sep 2010147 patients
Phase 3Completed

Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)

Start: Apr 2007Est. completion: Jun 200896 patients
Phase 3Completed

Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)

Start: Dec 2005Est. completion: Feb 200791 patients
Phase 3Completed

Subcutaneous Treatment With Icatibant for Acute Attacks of Hereditary Angioedema (HAE)

Start: Mar 2005Est. completion: Jul 200685 patients
Phase 3Completed

Subcutaneous Treatment With Icatibant for Acute Attacks of Hereditary Angioedema

Start: Dec 2004Est. completion: Jul 200684 patients
Phase 3Completed

Study to Evaluate Ecallantide in Paediatric Patients With Acute Attacks of Hereditary Angioedema

Start: Jun 20120
Phase 2/3Withdrawn

Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

Start: Jul 2017Est. completion: Jan 201958 patients
Phase 2Completed

Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema

Start: Aug 2016Est. completion: Aug 201775 patients
Phase 2Completed
NCT01756157TakedaCINRYZE with rHuPH20

Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema Attacks

Start: Feb 2013Est. completion: Sep 201347 patients
Phase 2Completed

CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

Start: Jun 2010Est. completion: Apr 20129 patients
Phase 2Completed

EDEMA2: Evaluation of DX-88's Effect in Mitigating Angioedema

Start: Nov 2003Est. completion: Jan 200677 patients
Phase 2Completed

A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants

Start: May 2014Est. completion: May 201538 patients
Phase 1Completed

Open-label, Single-arm Study to Assess the Pharmacokinetics, Safety, and Tolerability of a Single Subcutaneous Dose of Icatibant in Healthy Japanese Volunteers

Start: Feb 2014Est. completion: Feb 201412 patients
Phase 1Completed

Post Study Access of CSL312 (Garadacimab) for Pediatric Participants With Hereditary Angioedema Who Have Completed the CSL312_3003 Study

N/AAvailable
NCT07293364TakedaTechnochrom C1-INH Kit

A Study to Learn About the C1-Inhibitor Function as Diagnosis for Hereditary Angioedema

Start: Apr 2026Est. completion: Jan 2027514 patients
N/ANot Yet Recruiting

A Survey of Icatibant in Pediatric Participants With Hereditary Angioedema

Start: Aug 2022Est. completion: Oct 202532 patients
N/ACompleted

FIRAZYR General Drug Use-Results Survey (Japan)

Start: Nov 2018Est. completion: Jul 2024179 patients
N/ACompleted

Phase Legend

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

Key Insights

10 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 2,485 patients
5 companies competing in this space