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pegunigalsidase alfa-iwxj (Elfabrio)
Indications:
- enzyme replacement therapy (ERT) for Fabry disease
Dosage:
- titrate: 0.2, 1.0, 2.0 mg/kg, via intravenous infusion every other week
Injection: 2 mg/mL. 10 mL
Adverse effects:
- infusion-related reactions, hypersensitivity, asthenia
Mechanism of action:
- PEGylated, covalently crosslinked form of alpha-galactosidase A
Related
agalsidase beta (Fabrazyme, Replagal)
General
alpha-galactosidase A (ceramide trihexosidase, melibiase, GLA)
recombinant protein; chimer
metabolic agent (metabolic modifier)
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: 30834538 Clinical Trial.
- Wikipedia: Pegunigalsidase alfa
https://en.wikipedia.org/wiki/Pegunigalsidase_alfa