Drug class

Metabolic Disease Enzyme Replacement, Pompe Disease

Metabolic Disease Enzyme Replacement, Pompe Disease is a compendial and formulary therapeutic classification that encompasses exogenous enzyme therapies specifically designated to treat Pompe disease, also known as glycogen storage disease type II. Read more about Metabolic Disease Enzyme Replacement, Pompe Disease

4 drugs in this class 2 manufacturers

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About Metabolic Disease Enzyme Replacement, Pompe Disease.

Metabolic Disease Enzyme Replacement, Pompe Disease is a compendial and formulary therapeutic classification that encompasses exogenous enzyme therapies specifically designated to treat Pompe disease, also known as glycogen storage disease type II.

Agents in this class—such as recombinant human acid alpha-glucosidase formulations—function by supplying the deficient lysosomal enzyme, which is internalized by cells via mannose-6-phosphate receptors to degrade pathological intralysosomal glycogen deposits.

Consequently, this therapeutic category is primarily indicated to manage both infantile-onset and late-onset forms of Pompe disease, helping prevent or slow progressive cardiomyopathy, respiratory insufficiency, and skeletal muscle degradation.