Alglucosidase Alfa

A recombinant enzyme replacement therapy used to treat Pompe disease, a rare inherited condition where the absence of acid alpha-glucosidase allows glycogen to accumulate and destroy muscle cells. Regular infusions of this enzyme help break down the excess glycogen, improving muscle function, breathing, and survival particularly in the infantile-onset form.

Provides exogenous replacement of the deficient enzyme, restoring the metabolic pathway and clearing accumulated substrates from affected tissues.

Yes, Alglucosidase Alfa is an approved drug. It has reached clinical phase 4. It is classified as a Enzyme.