Elosulfase Alfa
Elosulfase Alfa Uses, Dosage, Side Effects, Food Interaction and all others data.
Elosulfase Alfa 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. Elosulfase Alfa 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.
| Trade Name | Elosulfase Alfa |
| 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 |
| Weight | 1mg/ml, |
| Type | Intravenous Solution, Intravenous |
| Formula | C5020H7588N1364O1418S34 |
| Weight | 110800.0 Da |
| Groups | Approved, Investigational |
| Therapeutic Class | |
| Manufacturer | |
| Available Country | United States |
| Last Updated: | September 19, 2023 at 7:00 am |
Uses
Elosulfase Alfa 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).
Elosulfase Alfa is also used to associated treatment for these conditions: Mucopolysaccharidosis Type IVA
How Elosulfase Alfa 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. Elosulfase Alfa 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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