Synonyms: BM-102; Galsulfasa; Galsulfasum; recombinant human arylsulfatase B; rhASB. N-Acetylgalactosamine 4-sulfatase.
Cyrillic synonym: Гальсульфас.

💊 Chemical information

CAS — 552858-79-4.
ATC — A1 6 AB08.
ATC Vet — QA16AB08.

💊 Profile

Galsulfase is recombinant human N-acetylgalactosamine 4-sulfatase used as enzyme replacement therapy in the treatment of mucopolysaccharidosis VI (see below). Galsulfase is given by intravenous infusion in a dose of 1 mg/kg once a week. Infusion reactions are common and patients should be pre-treated with antihistamines with or without antipyretics. Galsulfase should be reconstituted to a final volume of 250 mL in sodium chloride 0.9% and given using an infusion pump. The initial infusion rate should be 6 mL/hour for the first hour, which may then be increased to 80 mL/hour if well tolerated. The total infusion time should be at least 4 hours to minimise the risk of infusion reactions, but may be extended to up to 20 hours, or interrupted, if necessary, in the event of infusion reactions. Patients weighing 20 kg and under may be susceptible to fluid overload and a smaller infusion volume of 100 mL should be considered, in which case, the infusion rate should be decreased accordingly so that the total infusion time is not less than 4 hours.

Adverse effects.

1. Kim KH, et al. Successful management of difficult infusion-associated reactions in a young patient with mucopolysaccharidosis type VI receiving recombinant human arylsulfatase B (galsulfase [Naglazyme]). Abstract: Pediatrics 2008; 121: 609. Full version: 121/3/e714 (accessed 01/05/08)

Mucopolysaccharidosis VI.

Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) is a rare progressive disorder characterised by inherited deficiency of the enzyme N-acetylgalactosamine 4-sulfatase, which is necessary to catalyse the hydrolysis of the sulfate moiety of the glycosaminoglycan, dermatan sulfate. This results in accumulation of dermatan sulfate in the lysosomes producing widespread irreversible cellular and tissue damage, and organ dysfunction. There is a rapidly advancing form of the disease that presents in the first year of life characterised by short stature, skeletal and joint deformities, dysmorphic facial features, upper airway obstruction requiring tracheostomy, and recurrent ear infections. There is also a more slowly advancing form that progresses over many decades. Both forms result in significant morbidity and functional problems with a reduced lifespan.1 Treatment is supportive and symptomatic involving many body systems; physical and occupational therapy is also necessary.1Haematopoietic stem-cell transplantation to supply the deficient enzyme is of benefit to some patients, although it is associated with significant morbidity and mortality.1 Enzyme replacement therapy with galsulfase has been reported to confer benefit with an acceptable safety profile.1,2
1. Giugliani R, et al. Management guidelines for mucopolysaccharidosis VI. Pediatrics 2007; 120: 405–18
2. Harmatz P, et al. Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebocontrolled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr 2006; 148: 533–9.

💊 Preparations

Proprietary Preparations

Cz.: Naglazyme; Fr.: Naglazyme; Port.: Naglazyme; USA: Naglazyme.
Published May 08, 2019.