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Hereditary Angioedema (HAE)

4
Pipeline Programs
7
Companies
25
Clinical Trials
4 recruiting
1
Approved Products

Pipeline by Development Stage

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

Drug Modality Breakdown

Peptide
480%
RNA Therapeutic
120%
+ 20 programs with unclassified modality

On Market (1)

Approved therapies currently available

Takeda
FIRAZYRApproved
icatibant acetate
Takeda
subcutaneous2011
56M Part D

Competitive Landscape

7 companies ranked by most advanced pipeline stage

Takeda
TakedaTOKYO, Japan
14 programs
1
FirazyrN/A1 trial
Technochrom C1-INH KitN/A1 trial
DX-2930PHASE_11 trial
FIRAZYR(Icatibant)PHASE_11 trial
CINRYZEPHASE_23 trials
+9 more programs
Active Trials
NCT04057131Completed179Est. Jul 2024
NCT07293364Not Yet Recruiting514Est. Jan 2027
NCT02093923Completed38Est. May 2015
+13 more trials
CSL Seqirus
CSL SeqirusUK - Maidenhead
2 programs
1
CSL312Phase 3
CSL312N/A
Astria Therapeutics
2 programs
2
navenibartPhase 31 trial
navenibartPhase 31 trial
Active Trials
NCT07204938Enrolling By Invitation145Est. Dec 2031
NCT06842823Recruiting145Est. Sep 2027
CSL Behring
CSL BehringIL - Bradley
2 programs
CSL312N/A1 trial
CSL312PHASE_31 trial
Active Trials
NCT07159464Available
NCT05819775Completed22Est. Nov 2025
BioCryst Pharmaceuticals
2 programs
BCX7353PHASE_21 trial
BCX7353PHASE_21 trial
Active Trials
NCT02870972Completed75Est. Aug 2017
NCT03240133Completed58Est. Jan 2019
Pharvaris
PharvarisNetherlands - Leiden
2 programs
DeucrictibantPHASE_31 trial
DeucrictibantPHASE_31 trial
Active Trials
NCT06679881Recruiting170Est. Dec 2028
NCT06669754Active Not Recruiting81Est. Aug 2026
Ionis Pharmaceuticals
1 program
DonidalorsenPHASE_3RNA Therapeutic1 trial
Active Trials
NCT07298447Recruiting20Est. Jun 2029

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Ionis PharmaceuticalsDonidalorsen
Astria Therapeuticsnavenibart
Astria Therapeuticsnavenibart
PharvarisDeucrictibant
PharvarisDeucrictibant
CSL BehringCSL312
TakedaCINRYZE 500 U
TakedaCINRYZE
TakedaDX-2930 - 300mg/2wk
TakedaCINRYZE 500
Takedaecallantide
Takedaecallantide
Takedaecallantide
Takedaecallantide
TakedaCINRYZE

Showing 15 of 24 trials with date data

Clinical Trials (25)

Total enrollment: 2,238 patients across 25 trials

Donidalorsen Treatment in Children With Hereditary Angioedema

Start: Jul 2026Est. completion: Jun 202920 patients
Phase 3Recruiting

A Long-Term Study of Navenibart in Participants With Hereditary Angioedema

Start: Oct 2025Est. completion: Dec 2031145 patients
Phase 3Enrolling By Invitation

A Study of Navenibart in Participants With Hereditary Angioedema

Start: Mar 2025Est. completion: Sep 2027145 patients
Phase 3Recruiting

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
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

A Multicenter Study to Evaluate the Efficacy and Safety of Cinryze® for the Treatment of Acute Antibody-mediated Rejection in Participants With Kidney Transplant

Start: May 2016Est. completion: May 201939 patients
Phase 3Terminated
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

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

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

Start: Apr 2007Est. completion: Jun 200896 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 and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)

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

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

Start: Jun 20120
Phase 2/3Withdrawn

Complement Inhibition: Attacking the Overshooting Inflammation @Fter Subarachnoid Hemorrhage (CIAO@SAH)

Start: Nov 2024Est. completion: Mar 2027128 patients
Phase 2Recruiting

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

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

3 late-stage (Phase 3) programs, potential near-term approvals
4 actively recruiting trials targeting 2,238 patients
7 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.