Drug class

Metabolic Disease Enzyme Replacement, Fabry's Disease

Metabolic Disease Enzyme Replacement for Fabry's Disease is an administrative and clinical drug classification encompassing exogenous recombinant alpha-galactosidase A therapies, such as agalsidase beta and pegunigalsidase alfa. Read more about Metabolic Disease Enzyme Replacement, Fabry's Disease

3 drugs in this class 3 manufacturers

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Drugs in Metabolic Disease Enzyme Replacement, Fabry's Disease

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About Metabolic Disease Enzyme Replacement, Fabry's Disease.

Metabolic Disease Enzyme Replacement for Fabry's Disease is an administrative and clinical drug classification encompassing exogenous recombinant alpha-galactosidase A therapies, such as agalsidase beta and pegunigalsidase alfa.

Agents in this class work by cellular uptake and lysosomal internalization, providing functional enzyme to catalyze the breakdown of glycosphingolipids, predominantly globotriaosylceramide.

This therapeutic class is indicated specifically for the management of Fabry disease, an X-linked lysosomal storage disorder, to prevent substrate accumulation and attenuate progressive multiorgan dysfunction across the renal, cardiovascular, and nervous systems.