Alglucosidase Alfa

Class: Enzymes
Chemical Name: [199-arginine,223-histidine]prepro-α-glucosidase (human)
Molecular Formula: C₄₇₅₈H₇₂₆₂N₁₂₇₄O₁₃₆₉S₃₅
CAS Number: 420794-05-0
Brands: Myozyme

Medically reviewed by Drugs.com on Mar 28, 2022. Written by ASHP.

Introduction

Biosynthetic (recombinant DNA origin) form of human acid α-glucosidase (GAA); enzyme that catalyzes the hydrolysis of α-1,4- and α-1,6-glycosidic linkages of lysosomal glycogen.

Uses for Alglucosidase Alfa

Pompe Disease

Management of infantile-onset Pompe disease (acid α-glucosidase [GAA] deficiency, glycogen storage disease type II [GSD II], glycogenosis type II, acid maltase deficiency); designated an orphan drug by the FDA for use in this condition.

Pompe disease is a rare progressively debilitating and often fatal inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA.

In the infantile-onset form, Pompe disease results in intralysosomal accumulation of glycogen in various tissues, particularly cardiac and skeletal muscle and the liver, resulting in the development of cardiomyopathy, progressive muscle weakness, hepatomegaly, and impaired respiratory function; death secondary to cardiorespiratory failure usually occurs in the first year of life.

Alglucosidase alfa improves ventilator-free survival in patients with infantile-onset Pompe disease as compared with an untreated historical control group.

Safety and efficacy in patients with forms of Pompe disease other than infantile-onset not established.

Appears to have strongest and most consistent therapeutic effect on the cardiorespiratory manifestations of Pompe disease; effects on motor function appear highly dependent on patient’s condition at start of treatment. Effect of treatment on motor function over time unknown.

Alglucosidase Alfa Dosage and Administration

General

• May administer antipyretics and/or antihistamines prior to alglucosidase alfa infusion.

• If a severe reaction occurs, consider immediate discontinuance of the infusion and initiate appropriate medical treatment.

• Use caution when readministering alglucosidase alfa in patients who have experienced infusion reactions.

Administration

Administer by IV infusion.

IV Administration

Administer by IV infusion using an infusion pump and a 0.2-µm low-protein-binding inline filter.

Alglucosidase alfa particles (typically less than 10 in a vial) may be present in reconstituted alglucosidase alfa solutions in the form of thin white strands or translucent fibers subsequent to the initial inspection or following dilution for infusion; studies have shown that these particles are removed via inline filtration and do not have a detectable effect on the purity or strength of alglucosidase alfa solutions. Do not use solution if opaque particles or discoloration are observed immediately following reconstitution of the drug.

Do not infuse alglucosidase alfa infusions simultaneously through the same IV line with other ...