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Peer-reviewedCC BY-NCClinical and translational science

Garadacimab in Hereditary Angioedema: Modeling Suggests Sustained Improvements in Attack-Free Probability and Quality of Life Over 5 Years.

  • Philip H Li1
  • F Ida Hsu2
  • Emel Aygören-Pürsün3
  • William R Lumry4
  • Jason Raasch5
  • Ingo Pragst6
  • Yinglei Li7
  • Chiara Nenci8
  • Marco Campioni9
  • Markus Magerl10,11
11 affiliations
  1. 1Division of Rheumatology and Clinical Immunology, Department of Medicine, Queen Mary Hospital, University of Hong Kong, Hong Kong, China.
  2. 2Section of Rheumatology, Allergy & Immunology, Department of Medicine, Yale University School of Medicine, New Haven, Connecticut, USA.
  3. 3University Hospital Frankfurt, Department for Children and Adolescents, Goethe University Frankfurt, Frankfurt, Germany.
  4. 4AARA Research Center, Dallas, Texas, USA.
  5. 5Midwest Immunology Clinic, Plymouth, Minnesota, USA.
  6. 6CSL Innovation GmbH, Marburg, Germany.
  7. 7CSL Behring, King of Prussia, Pennsylvania, USA.
  8. 8CSL Behring AG, Bern, Switzerland.
  9. 9CSL Vifor Pharma Management Ltd., Zurich, Switzerland.
  10. 10Charité - University Medical Center Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität Zu Berlin, Institute of Allergology, Berlin, Germany.
  11. 11Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin, Germany.
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Abstract

Hereditary angioedema (HAE) attacks are recurrent, debilitating, and potentially life-threatening. Across the clinical program, garadacimab long-term prophylaxis demonstrated early onset and durable, sustained protection against HAE attacks and clinically meaningful improvements in health-related quality of life (HRQoL). HAE treatment goals include total disease control (attack-free) and normalization of life. Predicting treatment effect may aid shared decision-making and choice of long-term prophylaxis. Here, we report an attack-free probability model predicting garadacimab treatment effect beyond available Phase 3 clinical data and explore the relationship between HRQoL and attack-free duration. A generalized mixed-effect Poisson model was developed and validated with data from the garadacimab pivotal Phase 3 (VANGUARD) and Phase 3 open-label extension studies. Efficacy variables, including attack-free probabilities, were predicted for simulated populations of garadacimab-treated patients and assessed for goodness of fit. Analysis of covariance was used to assess the relationship between Angioedema Quality of Life questionnaire scores and attack-free duration. Model-predicted efficacy variables adequately reflected observed clinical data with high goodness of fit (Nakagawa-Schielzeth conditional R²: 74.3%). Following a 1-month attack-free period after garadacimab initiation, patients had a 60% cumulative probability of remaining attack-free up to Month 25 and 49% up to 5 years. HRQoL improved with increasing duration of attack-free periods, reaching minimal to no impairment after 6 months. This attack-free probability model suggests long-term effects of garadacimab over a 5-year treatment period. Patients treated with garadacimab have the potential to live attack-free for long periods of time, with substantial HRQoL improvements.

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Cite

BibTeXRIS
BibTeX
@article{li2026garadacimab,
  title = {{Garadacimab in Hereditary Angioedema: Modeling Suggests Sustained Improvements in Attack-Free Probability and Quality of Life Over 5 Years.}},
  author = {Philip H Li and F Ida Hsu and Emel Aygören-Pürsün and William R Lumry and Jason Raasch and Ingo Pragst and Yinglei Li and Chiara Nenci and Marco Campioni and Markus Magerl},
  journal = {Clinical and translational science},
  year = {2026},
  month = oct,
  doi = {10.1111/cts.70737},
  url = {https://europepmc.org/article/MED/42852816},
  note = {CC BY-NC},
}
RIS
TY  - JOUR
TI  - Garadacimab in Hereditary Angioedema: Modeling Suggests Sustained Improvements in Attack-Free Probability and Quality of Life Over 5 Years.
AU  - Philip H Li
AU  - F Ida Hsu
AU  - Emel Aygören-Pürsün
AU  - William R Lumry
AU  - Jason Raasch
AU  - Ingo Pragst
AU  - Yinglei Li
AU  - Chiara Nenci
AU  - Marco Campioni
AU  - Markus Magerl
PY  - 2026
DA  - 2026/10/01
JO  - Clinical and translational science
DO  - 10.1111/cts.70737
UR  - https://europepmc.org/article/MED/42852816
AB  - Hereditary angioedema (HAE) attacks are recurrent, debilitating, and potentially life-threatening. Across the clinical program, garadacimab long-term prophylaxis demonstrated early onset and durable, sustained protection against HAE attacks and clinically meaningful improvements in health-related quality of life (HRQoL). HAE treatment goals include total disease control (attack-free) and normalization of life. Predicting treatment effect may aid shared decision-making and choice of long-term prophylaxis. Here, we report an attack-free probability model predicting garadacimab treatment effect beyond available Phase 3 clinical data and explore the relationship between HRQoL and attack-free duration. A generalized mixed-effect Poisson model was developed and validated with data from the garadacimab pivotal Phase 3 (VANGUARD) and Phase 3 open-label extension studies. Efficacy variables, including attack-free probabilities, were predicted for simulated populations of garadacimab-treated patients and assessed for goodness of fit. Analysis of covariance was used to assess the relationship between Angioedema Quality of Life questionnaire scores and attack-free duration. Model-predicted efficacy variables adequately reflected observed clinical data with high goodness of fit (Nakagawa-Schielzeth conditional R²: 74.3%). Following a 1-month attack-free period after garadacimab initiation, patients had a 60% cumulative probability of remaining attack-free up to Month 25 and 49% up to 5 years. HRQoL improved with increasing duration of attack-free periods, reaching minimal to no impairment after 6 months. This attack-free probability model suggests long-term effects of garadacimab over a 5-year treatment period. Patients treated with garadacimab have the potential to live attack-free for long periods of time, with substantial HRQoL improvements.
SN  - 1752-8054
SN  - 1752-8062
ER  -