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pegunigalsidase alfa
- Development stage
- Approved
- Lead developer
- Chiesi
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, PEGylated Peptides → Modified Peptides → Peptides
- Administration
- Intravenous
01
Overview
Pegunigalsidase alfa is a pegylated recombinant form of human alpha-galactosidase A, developed as an enzyme replacement therapy for Fabry disease, a rare genetic disorder caused by deficiency of the lysosomal enzyme alpha-galactosidase A. It is produced in genetically modified Nicotiana tabacum (tobacco) plant cells using a plant cell-based protein expression system, resulting in distinct glycosylation patterns compared to other recombinant forms. Pegunigalsidase alfa is chemically modified with polyethylene glycol (PEG), which increases its stability and plasma half-life, allowing for less frequent dosing and potentially reduced immunogenicity. The drug acts by supplementing or replacing deficient alpha-galactosidase A activity, catalyzing the hydrolysis of terminal alpha-galactosyl moieties from oligosaccharides and polysaccharides within lysosomes. This reduces the accumulation of globotriaosylceramide (Gb3) and globotriaosylsphingosine (lyso-Gb3), key substrates implicated in Fabry disease pathology[1][2][4][5][6][8].
- Brand names
- Elfabrio
- Other names
- pegunigalsidase alfa-iwxj
02
Targets
Gb3 (Globotriaosylceramide)
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