A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

Exclusion Criteria16

• Contraindication to magnetic resonance imaging (MRI) including known history of hypersensitivity to gadolinium contrast agent that is not managed by the use of premedication.
• Pregnant at the time of enrolment.
• Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician, could interfere with the ability to participate in the study.
• Active participation in any interventional study for Fabry disease
• Treatment regimen at the time of enrolment in the study is different from the approved 1mg/kg every two weeks (note if regimen subsequently changes during the prospective part of the study, the participants can remain in the study)
• Prior participation in a pegunigalsidase alfa trial using a dose of 2 mg/kg administered every 4 weeks.
• Cardiac Cohort:
• History of acute myocardial infarction or congestive heart failure with reduced left ventricular (LV) ejection fraction of less than 35%.
• Cerebral vascular accident (CVA) in the prior 6 months.
• Chronic liver cirrhosis.
• FD-unrelated heart disease (e.g., scarring due to myocardial infarction, symptomatic occlusive coronary artery disease, moderate valvular heart disease not thought to be Fabry related).
• The participant is or has been treated with any investigational drug for Fabry disease within 6 months of study start or investigational gene therapy for Fabry disease at any time point in the past.
• Severe cardiac fibrosis defined as more than 3 segments that each have >50% fibrosis upon late gadolinium enhancement cMRI at any prior cMRI.
• Naïve Cohort:
• Prior exposure to a FD therapy (Replagal®, Fabrazyme®, and Galafold®) at any time point.
• Severe cardiac fibrosis defined as more than 3 segments that each have >50% fibrosis upon late gadolinium enhancement cMRI on any prior cMRI