Naglazyme (BioMarin Pharmaceuticals): Full Drug Profile

Naglazyme (BioMarin Pharmaceuticals) - General Information

Galsufase is a variant form of the polymorphic human enzyme N-acetylgalactosamine 4-sulfatase of recombinant DNA origin. Naglazyme (BioMarin Pharmaceuticals) is a glycoprotein with a molecular weight of approximately 56 kD. The recombinant protein is comprised of 495 amino acids and contains six asparagine-linked glycosylation sites, four of which carry a bis mannose-6-phosphate manose7 oligosaccharide for specific cellular recognition. Post-translational modification of Cys53 produces the catalytic amino acid residue Ca-formylglycine, which is required for enzyme activity and is conserved in all members of the sulfatase enzyme family.

Pharmacology of Naglazyme (BioMarin Pharmaceuticals)

Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of GAG. Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) is characterized by the absence or marked reduction in N-acetylgalactosamine 4-sulfatase. The sulfatase activity deficiency results in the accumulation of the GAG substrate dermatan sulfate, throughout the body. This accumulation leads to widespread cellular, tissue, and organ dysfunction. Naglazyme (BioMarin Pharmaceuticals) is intended to provide an exogenous enzyme that will be taken up into lysosomes and increase the catabolism of GAG. Naglazyme (BioMarin Pharmaceuticals) uptake by cells into lysosomes is most likely mediated by the binding of mannose-6-phosphate-terminated oligosaccharide chains of galsulfase to specific mannose-6-phosphate receptors.

Naglazyme (BioMarin Pharmaceuticals) for patients

Patients should be informed that a Clinical Surveilance Program has been established in order to better understand the variability and progression of the disease in the population as a whole, and to monitor and evaluate long-term treatment effects of NAGLAZYME. The Clinical Surveillance Program wil1 also monitor the effect of NAGLAZYME on pregnant women and their offspring, and determine if NAGLAZYME is excreted in breast milk. Patients should be encouraged to participate and advised that their participation is voluntary and may involve long-term follow-up. For more information, visit www.MPSVLcom/CSP or call (866) 906-6100.

Naglazyme (BioMarin Pharmaceuticals) Interactions

No formal drug interaction studies have been conducted.

Naglazyme (BioMarin Pharmaceuticals) Contraindications

None known.

Additional information about Naglazyme (BioMarin Pharmaceuticals)

Naglazyme (BioMarin Pharmaceuticals) Indication: For the treatment of adults and children with Mucopolysaccharidosis VI. Mechanism Of Action: Naglazyme (BioMarin Pharmaceuticals) supplies recombinant-engineered galsulfase, a normal variant form of the polymorphic human enzyme, N-acetylgalactosamine 4-sulfatase. It is a lysosomal hydrolase that catalyzes the cleavage of the sulfate ester from terminal N-acetylgalactosamine 4-sulfate residues of GAG chondroitin 4-sulfate and dermatan sulfate. Increased catabolism of GAG in turn reduces systemic dermatan sulfate accumulation, thereby reducing the primary symptoms of MPS VI. Drug Interactions: Not Available Food Interactions: Not Available Generic Name: Galsulfase Synonyms: Not Available Drug Category: Enzyme Replacement Agents Drug Type: Biotech; Approved Other Brand Names containing Galsulfase: Naglazyme (BioMarin Pharmaceuticals); Absorption: Not Available Toxicity (Overdose): There is no experience with overdose of galsulfase. Protein Binding: Not Available Biotransformation: Not Available Half Life: 9 (6 to 21) minutes during the first week of treatment, 26 (8 to 40) minutes by the 24th week. Dosage Forms of Naglazyme (BioMarin Pharmaceuticals): Solution Intravenous drip Chemical IUPAC Name: Not Available Chemical Formula: C2534H3851N691O719S16 Galsulfase on Wikipedia: https://en.wikipedia.org/wiki/Galsulfase Organisms Affected: Humans and other mammals