Alglucosidase Alfa

(USAN, rINN)
Synonyms: Alglucosidasa alfa; Alglucosidasum Alfa; rhGAA.
Cyrillic synonym: Альглюкозидаза Альфа.

💊 Chemical information

CAS — 420784-05-0.
ATC — A16AB07.
ATC Vet — QA16AB07.

💊 Profile

Alglucosidase alfa is a recombinant form of human acid alpha glucosidase given as enzyme replacement therapy for the treatment of the lysosomal storage disease Pompe disease (glycogen storage disease type II). This is a rare fatal autosomal recessive disorder caused by a deficiency of acid α-glucosidase, which cleaves α-1,4- and α-1,6-glucosidic linkages in lysosomal glycogen to liberate glucose. Glycogen accumulation results in progressive myopathy, especially of the skeletal muscles and heart. Alglucosidase alfa is given intravenously using an infusion pump in doses of 20 mg/kg once every 2 weeks. The total volume of fluid, which is determined by the patient’s body-weight, should be infused over about 4 hours. The infusion rate should be increased gradually: the initial rate should not exceed 1 mg/kg per hour; once the patient can tolerate this rate, it may be increased every 30 minutes by 2 mg/kg per hour with monitoring of vital signs before each increase; the maximum infusion rate is 7 mg/kg per hour. Infusion reactions are common with alglucosidase alfa; symptoms may resolve on decreasing the infusion rate, temporarily stopping the infusion, and/or use of antihistamines and/or antipyretics, which may also be given as pre-treatment. Severe reactions may require stopping the infusion immediately. Serious hypersensitivity reactions, including anaphylactic shock, have also been reported during infusion of alglucosidase alfa.
1. Amalfitano A, et al. Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial. Genet Med 2001; 3: 132–8
2. Van den Hout JM, et al. Enzyme therapy for Pompe disease with recombinant human alpha-glucosidase from rabbit milk. J Inherit Metab Dis 2001; 24: 266–74
3. Kishnani PS, Howell RR. Pompe disease in infants and children. J Pediatr 2004; 144 (suppl): S35–S43
4. Hunley TE, et al. Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease. Abstract: Pediatrics 2004; 114: 1080. Full version: http://www.pediatrics.org/ cgi/content/full/114/4/e532 (accessed 17/01/06
5. Kishnani PS, et al. Chinese hamster ovary cell-derived recombinant human acid α-glucosidase in infantile-onset Pompe disease. J Pediatr 2006; 149: 89–97
6. van der Beek NA, et al. Pompe disease (glycogen storage disease type II): clinical features and enzyme replacement therapy. Acta Neurol Belg 2006; 106: 82–6
7. Kishnani PS, et al. Recombinant human acid α-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology 2007; 68: 99–109
8. Fukuda T, et al. Acid alpha-glucosidase deficiency (Pompe disease). Curr Neurol Neurosci Rep 2007; 7: 71–7
9. Rossi M, et al. Long-term enzyme replacement therapy for Pompe disease with recombinant human alpha-glucosidase derived from Chinese hamster ovary cells. J Child Neurol 2007; 22: 565–73.

💊 Preparations

Proprietary Preparations

Cz.: Myozyme; Fr.: Myozyme; Port.: Myozyme; UK: Myozyme; USA: Myozyme. Multi-ingredient: Austral.: Digestaid; Canad.: Digesta.
Published October 17, 2018.