HAE in disease context refers to hereditary angioedema, a rare inherited disorder causing recurrent attacks of severe, non-histaminergic swelling involving skin, gastrointestinal tract, and airway. Attacks can be life-threatening when the larynx is affected. The condition is commonly related to C1-inhibitor pathway dysfunction and bradykinin-mediated edema.
Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.
Phase 2 data for BW-20805 showed up to 100% reduction in HAE attacks with dosing intervals up to 6 months, while onvuzosiran (ADX-324) advances to Phase 3 trials with semi-annual dosing targeting prekallikrein.
The FDA removed the clinical hold on Intellia Therapeutics' MAGNITUDE-2 Phase 3 trial of nexiguran ziclumeran for ATTRv-PN, allowing patient enrollment and dosing to resume with enhanced safety monitoring.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07559630 |
Rectal Arterial Embolization vs Band Ligation for the Treatment of Internal hemOrrhoidS |
RECRUITING | NA |
| NCT07428499 |
Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE) |
RECRUITING | PHASE3 |
| NCT07179601 |
Hemorrhoidal Artery Embolization: Longitudinal Impact On Symptoms (HELIOS) |
RECRUITING | NA |
| NCT06960213 |
STOP-HAE: A Phase 3 Study of ADX-324 in HAE |
RECRUITING | PHASE3 |
| NCT05121376 |
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema |
ACTIVE_NOT_RECRUITING | PHASE1/PHASE2 |
| NCT04933721 |
Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT04428632 |
Oral Berotralstat Expanded Access Program |
APPROVED_FOR_MARKETING | |
| NCT03485911 |
Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE |
COMPLETED | PHASE3 |
| NCT03472040 |
A Long Term Safety Study of BCX7353 in Hereditary Angioedema |
COMPLETED | PHASE2/PHASE3 |
| NCT03029728 |
Biomarker for Hereditary AngioEdema Disease |
COMPLETED |
