pegunigalsidase alfa-iwxj (Elfabrio)
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Indications
- enzyme replacement therapy (ERT) for Fabry disease
Dosage
- titrate: 0.2, 1.0, 2.0 mg/kg, via intravenous infusion every other week
Adverse effects
- infusion-related reactions, hypersensitivity, asthenia
Mechanism of action
- PEGylated, covalently crosslinked form of alpha-galactosidase A
More general terms
- alpha-galactosidase A (ceramide trihexosidase, melibiase, GLA)
- recombinant protein; chimer
- metabolic agent (metabolic modifier)
Additional terms
References
- ↑ Schiffmann R, Goker-Alpan O, Holida M et al Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial. J Inherit Metab Dis. 2019 May;42(3):534-544. PMID: https://www.ncbi.nlm.nih.gov/pubmed/30834538 Clinical Trial.
- ↑ Wikipedia: Pegunigalsidase alfa https://en.wikipedia.org/wiki/Pegunigalsidase_alfa