Floatz Rating
BB67/100
Confidence
Indicative
Sponsor
Ionis Pharmaceuticals, Inc.
Modality
UNKNOWN
Development Phase
Phase 3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
56High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT07298447CT.gov Donidalorsen Treatment in Children With Hereditary Angioedema | — | Recruiting | — | — | — |
NCT05392114CT.gov A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) | — | Active Not Recruiting | — | — | — |
NCT05139810CT.gov OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE) | — | Completed | — | — | — |
NCT04307381CT.gov An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema | — | Completed | — | — | — |
NCT04030598CT.gov A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema | — | Completed | — | — | — |
NCT06415448CT.gov Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema | — | Available | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Hereditary Angioedema
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Donidalorsen (this asset) | Ionis Pharmaceuticals, Inc. | P3 | BB · 67 |
| Firazyr | Nang Kuang Pharmaceutical Co., Ltd. | P4 | A |
| Sebetralstat | KalVista Pharmaceuticals, Ltd. | P4 | A |
| Berotralstat | BioCryst Pharmaceuticals | P3 | A |
| Lanadelumab | Takeda | P3 | A |
| Garadacimab | CSL Behring | P4 | BBB |
| Ecallantide | NYU Langone Health | P3 | BBB |
| Deucrictibant | Institute for Asthma and Allergy | P3 | BBB |
| c1 esterase inhibitor [human] | Shire | P3 | BBB |
+28 more in the Hereditary Angioedema cohort
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Donidalorsen in Hereditary Angioedema. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/donidalorsen-hereditary-angioedema
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