==== Reference: Usmani SS, Bedi G, Samuel JS, Singh S, Kalra S, Kumar P, et al. (2017) THPdb: Database of FDA-approved peptide and protein therapeutics. PLoS ONE 12(7) e0181748.====

Detailed description page of THPdb

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Th1177 details
Primary information
ID1735
ThPP IDTh1177
Therapeutic Peptide/Protein NameElosulfase alfa
SequenceAPQPPNILLLLMDDMGWGDLGVYGEPSRETPLCSPSRAALLTGRLPIRNG view full sequnce in fasta
Functional ClassificationIa
Molecular Weight110800
Chemical FormulaC5020H7588N1364O1418S34
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half Lifeweek 0: 7.52 min week 22: 35.9 min
DescriptionElosulfase 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 Vimizimâ„¢. 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.
Indication/DiseaseVimizim is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
PharmacodynamicsAUC: 238 min x μg/mL, standard deviation 100.
Mechanism of ActionMucopolysaccharidoses 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.
ToxicityNA
MetabolismNA
AbsorptionCmax: 1.49 μg/mL, standard deviation 0.534.
Volume of Distribution396 mL/kg, standard deviation 316.
Clearance10.0 mL/min/kg. (standard deviation: 3.73).
CategoriesEnzymes; Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetN-acetylgalactosamine-6-sulfatase
Information of corresponding available drug in the market
Brand NameNA
CompanyNA
Brand DiscriptionNA
Prescribed forNA
Chemical NameNA
FormulationNA
Physcial AppearanceInjection, solution
Route of AdministrationIV
Recommended DosageNA
ContraindicationNA
Side EffectsNA
Useful Link
PubMed ID24958960
3-D StructureTh1177 (View) or (Download)
Primary information
ID1736
ThPP IDTh1177
Therapeutic Peptide/Protein NameElosulfase alfa
SequenceAPQPPNILLLLMDDMGWGDLGVYGEPSRETPLCSPSRAALLTGRLPIRNG view full sequnce in fasta
Functional ClassificationIa
Molecular Weight110800
Chemical FormulaC5020H7588N1364O1418S34
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half Lifeweek 0: 7.52 min week 22: 35.9 min
DescriptionElosulfase 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 Vimizimâ„¢. 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.
Indication/DiseaseVimizim is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
PharmacodynamicsAUC: 238 min x μg/mL, standard deviation 100.
Mechanism of ActionMucopolysaccharidoses 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.
ToxicityNA
MetabolismNA
AbsorptionCmax: 1.49 μg/mL, standard deviation 0.534.
Volume of Distribution397 mL/kg, standard deviation 316.
Clearance10.0 mL/min/kg. (standard deviation: 3.73).
CategoriesEnzymes; Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetN-acetylgalactosamine-6-sulfatase
Information of corresponding available drug in the market
Brand NameVimizim
CompanyBiomarin International Limited
Brand DiscriptionVimizim is a formulation of elosulfase alfa, which is a purified human enzyme produced by recombinant DNA technology in a Chinese hamster ovary cell line. Human N-acetylgalactosamine-6-sulfatase (EC 3.1.6.4) is a hydrolytic lysosomal glycosaminoglycan-specific enzyme that hydrolyzes sulfate from either galactose-6-sulfate or N-acetyl-galactosamine-6-sulfate on the non-reducing ends of the glycosaminoglycans keratan sulfate (KS) and chondroitin-6-sulfate (C6S).
Prescribed forVimizim (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
Chemical NameNA
Formulation1 mg
Physcial AppearanceSolution
Route of AdministrationIV
Recommended DosageThe 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. Pre-treatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to the start of the infusion
ContraindicationNA
Side EffectsAnaphylaxis and hypersensitivity reactions.
Useful Link
PubMed ID24958960
3-D StructureTh1177 (View) or (Download)
Primary information
ID1737
ThPP IDTh1177
Therapeutic Peptide/Protein NameElosulfase alfa
SequenceAPQPPNILLLLMDDMGWGDLGVYGEPSRETPLCSPSRAALLTGRLPIRNG view full sequnce in fasta
Functional ClassificationIa
Molecular Weight110800
Chemical FormulaC5020H7588N1364O1418S35
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half Lifeweek 0: 7.52 min week 22: 35.9 min
DescriptionElosulfase 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 Vimizimâ„¢. 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.
Indication/DiseaseVimizim is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
PharmacodynamicsAUC: 238 min x μg/mL, standard deviation 100.
Mechanism of ActionMucopolysaccharidoses 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.
ToxicityNA
MetabolismNA
AbsorptionCmax: 1.49 μg/mL, standard deviation 0.534.
Volume of Distribution398 mL/kg, standard deviation 316.
Clearance10.0 mL/min/kg. (standard deviation: 3.73).
CategoriesEnzymes; Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetN-acetylgalactosamine-6-sulfatase
Information of corresponding available drug in the market
Brand NameVimizim
CompanyBio Marin Pharmaceutical Inc.
Brand DiscriptionVimizim is a formulation of elosulfase alfa, which is a purified human enzyme produced by recombinant DNA technology in a Chinese hamster ovary cell line. Human N-acetylgalactosamine-6-sulfatase (EC 3.1.6.4) is a hydrolytic lysosomal glycosaminoglycan-specific enzyme that hydrolyzes sulfate from either galactose-6-sulfate or N-acetyl-galactosamine-6-sulfate on the non-reducing ends of the glycosaminoglycans keratan sulfate (KS) and chondroitin-6-sulfate (C6S).
Prescribed forVimizim (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
Chemical NameNA
Formulation5 mg/5mL
Physcial AppearanceInjection, solution
Route of AdministrationIV
Recommended DosageThe 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. Pre-treatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to the start of the infusion
ContraindicationNA
Side EffectsAnaphylaxis and hypersensitivity reactions.
Useful Link
PubMed ID24958960
3-D StructureTh1177 (View) or (Download)