Hereditary angioedema is a rare inherited disorder causing recurrent episodes of severe swelling of skin, gastrointestinal tract, and upper airway. The mechanism is typically bradykinin-mediated and commonly linked to C1 inhibitor pathway defects. Airway attacks can be life-threatening and require prompt recognition and specific therapy.
Swissmedic has authorized three orphan drugs for rare diseases: Breyanzi for mantle cell lymphoma, Alhemo for haemophilia A and B, and Ekterly for hereditary angioedema. The decisions include a new indication extension for Breyanzi, an expanded indication for Alhemo, and the initial authorization of Ekterly as part of a joint international regulatory initiative.
ADARx will present onvuzosiran Phase 1/2 data and Phase 3 STOP-HAE design at AAAAI and EAACI 2026. The investigational siRNA for hereditary angioedema has FDA Orphan Drug Designation.
January and March 2026 drug pipeline updates included Phase 3 results, Fast Track designations and Priority Review decisions across multiple disease areas. Mavacamten, teplizumab-mzwv, anifrolumab, AXS-05, gedatolisib and AFTX-201 were among the programs with notable milestones.
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.
The FDA approved 46 novel drugs in 2025, down from 50 in 2024. Small molecules accounted for 31 approvals (67%), with oncology leading at nine approvals. Large molecules contributed 15 approvals (33%), spanning ADCs, bispecifics, and subcutaneous delivery innovations.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07759141 |
EAP for Deucrictibant Immediate-release |
AVAILABLE | |
| NCT07654829 |
Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL) |
NOT_YET_RECRUITING | PHASE4 |
| NCT07216378 |
Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat |
AVAILABLE | |
| NCT07009262 |
A Study Observing US Patients With HAE Type I or II Who Take Icatibant to Treat HAE Attacks |
COMPLETED | |
| NCT06960213 |
STOP-HAE: A Phase 3 Study of ADX-324 in HAE |
RECRUITING | PHASE3 |
| NCT06806657 |
Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab |
COMPLETED | PHASE4 |
| NCT06690047 |
Treatment of Hereditary Angioedema Prodrome with Recombinant C1-esterase Inhibitor (Ruconest) |
COMPLETED | PHASE4 |
| NCT06634420 |
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE) |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT06628713 |
Treatment of Angioedema Attacks in Adolescent and Adult Patients 12 Years and Older With HAE Type I or II With Sebetralstat |
APPROVED_FOR_MARKETING | |
| NCT06573723 |
Institutional Registry of Rare Diseases |
RECRUITING |
