Pegunigalsidase-alfa may represent an advance in ERT for FD, based on its unique pharmacokinetics and apparent low immunogenicity. The objective of the study is to document long term data on treatment with pegunigalsidase-alfa under "real world" conditions. 60 patients with FD (therapy-naïve or pretreated with agalsidase-alfa or agalsidase-beta) will be recruited in 8 German Fabry centers. The treatment duration/patient will be 2 years. All patients will be followed-up by the above listed Fabry expert centers.
Pegunigalsidase-alfa, a novel PEGylated, covalently crosslinked form of α-galactosidase A developed as enzyme replacement therapy (ERT) for Fabry disease (FD), was designed to increase plasma half-life and reduce immunogenicity, thereby enhancing efficacy compared with available products. The rationale of the current project is that disease progression of patients with FD can be stabilized comparable to patients under current ERT, leading to a validation of the clinical phase 3-studies and a transfer of these previous outcomes to a nationwide "real world" designed study in Germany.
Study Type
OBSERVATIONAL
Enrollment
60
Standard of care
Fabry disease center Berlin - Charité - Universitätsmedizin Berlin
Berlin, Germany
Fabry disease center Cologne, Universitätsklinikum Köln
Cologne, Germany
Fabry disease center Hamburg, Universitätsklinikum Hamburg
Hamburg, Germany
Fabry disease center Hannover, Universitätsklinikum Hannover
Hanover, Germany
Fabry disease center Mainz, Universitätsmedizin Mainz
Mainz, Germany
Fachinternistische Gemeinschaftspraxis, Müllheim
Müllheim, Germany
Fabry disease center Münster, Universitätsklinikum Münster
Münster, Germany
Fabry disease center Würzburg, Universitätsklinikum Würzburg
Würzburg, Germany
eGFR
eGFR: Change in annualized eGFR slope compared with annualized eGFR slope before treatment start or switch.
Time frame: yearly
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