Recombinant Human N-acetylgalactosamine-6-sulfatase (rhGALNS)
Recombinant Human N-acetylgalactosamine-6-sulfatase (rhGALNS) Uses, Dosage, Side Effects, Food Interaction and all others data.
Recombinant Human N-acetylgalactosamine-6-sulfatase (rhGALNS) is a synthetic version of the enzyme N-acetylgalactosamine-6-sulfatase. It was approved by the FDA in 2014 for the treatment of Morquio syndrome. Recombinant Human N-acetylgalactosamine-6-sulfatase (rhGALNS) was developed by BioMarin Pharmaceutical Inc. and is marketed under the brand Vimizim™. The recommended dose is 2 mg per kg given intravenously over a minimum range of 3.5 to 4.5 hours, based on infusion volume, once every week.
AUC: 238 min x μg/mL, standard deviation 100.
Table Of contents
| Trade Name | Recombinant Human N-acetylgalactosamine-6-sulfatase (rhGALNS) |
| Generic | Elosulfase alfa |
| Elosulfase alfa Other Names | Chondroitin sulfatase, Chondroitinase, Chondrosulfatase, Elosulfase alfa, Recombinant human N-acetylgalactosamine-6-sulfatase, Recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS), rhGALNS |
| Type | |
| Formula | C5020H7588N1364O1418S34 |
| Weight | 110800.0 Da |
| Groups | Approved, Investigational |
| Therapeutic Class | |
| Manufacturer | |
| Available Country | |
| Last Updated: | September 19, 2023 at 7:00 am |
Uses
Recombinant Human N-acetylgalactosamine-6-sulfatase (rhGALNS) is a lysosomal glycosaminoglycan (GAG)-specific enzyme indicated as an enzyme replacement therapy for Mucopolysaccharidosis type IV A.
Vimizim is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
Recombinant Human N-acetylgalactosamine-6-sulfatase (rhGALNS) is also used to associated treatment for these conditions: Mucopolysaccharidosis Type IVA
How Recombinant Human N-acetylgalactosamine-6-sulfatase (rhGALNS) works
Mucopolysaccharidoses comprise a group of lysosomal storage disorders caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG). Mucopolysaccharidosis IVA (MPS IVA, Morquio A Syndrome) is characterized by the absence or marked reduction in N-acetylgalactosamine-6-sulfatase activity. The sulfatase activity deficiency results in the accumulation of the GAG substrates, KS and C6S, in the lysosomal compartment of cells throughout the body. The accumulation leads to widespread cellular, tissue, and organ dysfunction. Vimizim is intended to provide the exogenous enzyme N-acetylgalactosamine-6-sulfatase that will be taken up into the lysosomes and increase the catabolism of the GAGs KS and C6S. Recombinant Human N-acetylgalactosamine-6-sulfatase (rhGALNS) uptake by cells into lysosomes is mediated by the binding of mannose-6-phosphate-terminated oligosaccharide chains of elosulfase alfa to mannose-6-phosphate receptors.
In the absence of an animal disease model that recapitulates the human disease phenotype, elosulfase alfa pharmacological activity was evaluated using human primary chondrocytes from two MPS IVA patients. Treatment of MPS IVA chondrocytes with elosulfase alfa induced clearance of KS lysosomal storage from the chondrocytes.
Food Interaction
No interactions found.Volume of Distribution
396 mL/kg, standard deviation 316.
Elimination Route
Cmax: 1.49 μg/mL, standard deviation 0.534.
Half Life
week 0: 7.52 min week 22: 35.9 min
Clearance
10.0 mL/min/kg. (standard deviation: 3.73).
Innovators Monograph
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