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Th1166 details
Primary information
ID1693
ThPP IDTh1166
Therapeutic Peptide/Protein NameAsfotase Alfa
SequenceLVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTV view full sequnce in fasta
Functional ClassificationIa
Molecular Weight180000
Chemical FormulaC7108H11008N1968O2206S56
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half LifeApproximately 5 days.
DescriptionAsfotase Alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body’s ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015.
Indication/DiseaseIndicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
PharmacodynamicsPerinatal/infantile- and juvenile-onset HPP patients treated with Asfotase alfa had reductions in plasma TNSALP (tissue non-specific alkaline phosphatase) substrates, PPi and pyridoxal 5'-phosphate (PLP) within 6 to 12 weeks of treatment. Reductions in plasma PPi and PLP levels did not correlate with clinical outcomes. Bone biopsy data from perinatal/infantile-onset and juvenile-onset HPP patients treated with Asfotase alfa demonstrated decreases in osteoid volume and thickness indicating improved bone mineralization.
Mechanism of ActionHPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
ToxicityThere are no available human data on Asfotase Alfa use in pregnant women to inform a drug associated risk. In animal reproduction studies, Asfotase Alfa administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no evidence of fetotoxicity, embryolethality or teratogenicity at doses causing plasma exposures up to 21 and 24 times, respectively, the exposure at the recommended human dose.
MetabolismNA
AbsorptionNA
Volume of DistributionNA
ClearanceNA
CategoriesEnzymes Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetSphingosine 1-phosphate receptor 1; Pyrophosphate
Information of corresponding available drug in the market
Brand NameNA
CompanyNA
Brand DiscriptionNA
Prescribed forNA
Chemical NameNA
FormulationNA
Physcial AppearanceNA
Route of AdministrationNA
Recommended DosageNA
ContraindicationNA
Side EffectsHypersensitivity Reactions; Lipodystrophy; Ectopic Calcifications.
Useful Linkhttp://www.rxlist.com/strensiq-drug/side-effects-interactions.htm
PubMed ID26893260, 26529632, 22397652
3-D StructureTh1166 (View) or (Download)
Primary information
ID1694
ThPP IDTh1166
Therapeutic Peptide/Protein NameAsfotase Alfa
SequenceLVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTV view full sequnce in fasta
Functional ClassificationIa
Molecular Weight180001
Chemical FormulaC7108H11008N1968O2206S57
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half LifeApproximately 5 days.
DescriptionAsfotase Alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body’s ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015.
Indication/DiseaseIndicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
PharmacodynamicsPerinatal/infantile- and juvenile-onset HPP patients treated with Asfotase alfa had reductions in plasma TNSALP (tissue non-specific alkaline phosphatase) substrates, PPi and pyridoxal 5'-phosphate (PLP) within 6 to 12 weeks of treatment. Reductions in plasma PPi and PLP levels did not correlate with clinical outcomes. Bone biopsy data from perinatal/infantile-onset and juvenile-onset HPP patients treated with Asfotase alfa demonstrated decreases in osteoid volume and thickness indicating improved bone mineralization.
Mechanism of ActionHPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
ToxicityThere are no available human data on Asfotase Alfa use in pregnant women to inform a drug associated risk. In animal reproduction studies, Asfotase Alfa administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no evidence of fetotoxicity, embryolethality or teratogenicity at doses causing plasma exposures up to 21 and 24 times, respectively, the exposure at the recommended human dose.
MetabolismNA
AbsorptionNA
Volume of DistributionNA
ClearanceNA
CategoriesEnzymes Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetSphingosine 1-phosphate receptor 1; Pyrophosphate
Information of corresponding available drug in the market
Brand NameStrensiq
CompanyAlexion Pharma Ghbh
Brand DiscriptionNA
Prescribed forSTRENSIQâ„¢ is indicated for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP).
Chemical NameNA
Formulation100 mg
Physcial AppearanceSolution
Route of AdministrationSubcutaneous
Recommended DosageThe recommended dosage regimen of STRENSIQ for the treatment of perinatal/infantileonset HPP is 6 mg/kg per week administered subcutaneously as either: 2 mg/kg three times per week, or 1 mg/kg six times per week. Injection site reactions may limit the tolerability of the six times per week regimen
ContraindicationNA
Side EffectsHypersensitivity Reactions; Lipodystrophy; Ectopic Calcifications.
Useful Linkhttp://www.rxlist.com/strensiq-drug/side-effects-interactions.htm
PubMed ID26893260, 26529632, 22397653
3-D StructureTh1166 (View) or (Download)
Primary information
ID1695
ThPP IDTh1166
Therapeutic Peptide/Protein NameAsfotase Alfa
SequenceLVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTV view full sequnce in fasta
Functional ClassificationIa
Molecular Weight180002
Chemical FormulaC7108H11008N1968O2206S58
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half LifeApproximately 5 days.
DescriptionAsfotase Alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body’s ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015.
Indication/DiseaseIndicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
PharmacodynamicsPerinatal/infantile- and juvenile-onset HPP patients treated with Asfotase alfa had reductions in plasma TNSALP (tissue non-specific alkaline phosphatase) substrates, PPi and pyridoxal 5'-phosphate (PLP) within 6 to 12 weeks of treatment. Reductions in plasma PPi and PLP levels did not correlate with clinical outcomes. Bone biopsy data from perinatal/infantile-onset and juvenile-onset HPP patients treated with Asfotase alfa demonstrated decreases in osteoid volume and thickness indicating improved bone mineralization.
Mechanism of ActionHPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
ToxicityThere are no available human data on Asfotase Alfa use in pregnant women to inform a drug associated risk. In animal reproduction studies, Asfotase Alfa administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no evidence of fetotoxicity, embryolethality or teratogenicity at doses causing plasma exposures up to 21 and 24 times, respectively, the exposure at the recommended human dose.
MetabolismNA
AbsorptionNA
Volume of DistributionNA
ClearanceNA
CategoriesEnzymes Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetSphingosine 1-phosphate receptor 1; Pyrophosphate
Information of corresponding available drug in the market
Brand NameStrensiq
CompanyAlexion Pharmaceuticals Inc.
Brand DiscriptionNA
Prescribed forSTRENSIQâ„¢ is indicated for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP).
Chemical NameNA
Formulation18 mg/.45mL
Physcial AppearanceSolution
Route of AdministrationSubcutaneous
Recommended DosageThe recommended dosage regimen of STRENSIQ for the treatment of perinatal/infantileonset HPP is 6 mg/kg per week administered subcutaneously as either: 2 mg/kg three times per week, or 1 mg/kg six times per week. Injection site reactions may limit the tolerability of the six times per week regimen
ContraindicationNA
Side EffectsHypersensitivity Reactions; Lipodystrophy; Ectopic Calcifications.
Useful Linkhttp://www.rxlist.com/strensiq-drug/side-effects-interactions.htm
PubMed ID26893260, 26529632, 22397654
3-D StructureTh1166 (View) or (Download)
Primary information
ID1696
ThPP IDTh1166
Therapeutic Peptide/Protein NameAsfotase Alfa
SequenceLVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTV view full sequnce in fasta
Functional ClassificationIa
Molecular Weight180003
Chemical FormulaC7108H11008N1968O2206S59
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half LifeApproximately 5 days.
DescriptionAsfotase Alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body’s ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015.
Indication/DiseaseIndicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
PharmacodynamicsPerinatal/infantile- and juvenile-onset HPP patients treated with Asfotase alfa had reductions in plasma TNSALP (tissue non-specific alkaline phosphatase) substrates, PPi and pyridoxal 5'-phosphate (PLP) within 6 to 12 weeks of treatment. Reductions in plasma PPi and PLP levels did not correlate with clinical outcomes. Bone biopsy data from perinatal/infantile-onset and juvenile-onset HPP patients treated with Asfotase alfa demonstrated decreases in osteoid volume and thickness indicating improved bone mineralization.
Mechanism of ActionHPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
ToxicityThere are no available human data on Asfotase Alfa use in pregnant women to inform a drug associated risk. In animal reproduction studies, Asfotase Alfa administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no evidence of fetotoxicity, embryolethality or teratogenicity at doses causing plasma exposures up to 21 and 24 times, respectively, the exposure at the recommended human dose.
MetabolismNA
AbsorptionNA
Volume of DistributionNA
ClearanceNA
CategoriesEnzymes Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetSphingosine 1-phosphate receptor 1; Pyrophosphate
Information of corresponding available drug in the market
Brand NameStrensiq
CompanyAlexion Pharma Ghbh
Brand DiscriptionNA
Prescribed forSTRENSIQâ„¢ is indicated for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP).
Chemical NameNA
Formulation40 mg
Physcial AppearanceSolution
Route of AdministrationSubcutaneous
Recommended DosageThe recommended dosage regimen of STRENSIQ for the treatment of perinatal/infantileonset HPP is 6 mg/kg per week administered subcutaneously as either: 2 mg/kg three times per week, or 1 mg/kg six times per week. Injection site reactions may limit the tolerability of the six times per week regimen
ContraindicationNA
Side EffectsHypersensitivity Reactions; Lipodystrophy; Ectopic Calcifications.
Useful Linkhttp://www.rxlist.com/strensiq-drug/side-effects-interactions.htm
PubMed ID26893260, 26529632, 22397655
3-D StructureTh1166 (View) or (Download)
Primary information
ID1697
ThPP IDTh1166
Therapeutic Peptide/Protein NameAsfotase Alfa
SequenceLVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTV view full sequnce in fasta
Functional ClassificationIa
Molecular Weight180004
Chemical FormulaC7108H11008N1968O2206S60
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half LifeApproximately 5 days.
DescriptionAsfotase Alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body’s ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015.
Indication/DiseaseIndicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
PharmacodynamicsPerinatal/infantile- and juvenile-onset HPP patients treated with Asfotase alfa had reductions in plasma TNSALP (tissue non-specific alkaline phosphatase) substrates, PPi and pyridoxal 5'-phosphate (PLP) within 6 to 12 weeks of treatment. Reductions in plasma PPi and PLP levels did not correlate with clinical outcomes. Bone biopsy data from perinatal/infantile-onset and juvenile-onset HPP patients treated with Asfotase alfa demonstrated decreases in osteoid volume and thickness indicating improved bone mineralization.
Mechanism of ActionHPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
ToxicityThere are no available human data on Asfotase Alfa use in pregnant women to inform a drug associated risk. In animal reproduction studies, Asfotase Alfa administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no evidence of fetotoxicity, embryolethality or teratogenicity at doses causing plasma exposures up to 21 and 24 times, respectively, the exposure at the recommended human dose.
MetabolismNA
AbsorptionNA
Volume of DistributionNA
ClearanceNA
CategoriesEnzymes Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetSphingosine 1-phosphate receptor 1; Pyrophosphate
Information of corresponding available drug in the market
Brand NameStrensiq
CompanyAlexion Pharma Ghbh
Brand DiscriptionNA
Prescribed forSTRENSIQâ„¢ is indicated for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP).
Chemical NameNA
Formulation40 mg
Physcial AppearanceSolution
Route of AdministrationSubcutaneous
Recommended DosageThe recommended dosage regimen of STRENSIQ for the treatment of perinatal/infantileonset HPP is 6 mg/kg per week administered subcutaneously as either: 2 mg/kg three times per week, or 1 mg/kg six times per week. Injection site reactions may limit the tolerability of the six times per week regimen
ContraindicationNA
Side EffectsHypersensitivity Reactions; Lipodystrophy; Ectopic Calcifications.
Useful Linkhttp://www.rxlist.com/strensiq-drug/side-effects-interactions.htm
PubMed ID26893260, 26529632, 22397656
3-D StructureTh1166 (View) or (Download)
Primary information
ID1698
ThPP IDTh1166
Therapeutic Peptide/Protein NameAsfotase Alfa
SequenceLVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTV view full sequnce in fasta
Functional ClassificationIa
Molecular Weight180005
Chemical FormulaC7108H11008N1968O2206S61
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half LifeApproximately 5 days.
DescriptionAsfotase Alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body’s ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015.
Indication/DiseaseIndicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
PharmacodynamicsPerinatal/infantile- and juvenile-onset HPP patients treated with Asfotase alfa had reductions in plasma TNSALP (tissue non-specific alkaline phosphatase) substrates, PPi and pyridoxal 5'-phosphate (PLP) within 6 to 12 weeks of treatment. Reductions in plasma PPi and PLP levels did not correlate with clinical outcomes. Bone biopsy data from perinatal/infantile-onset and juvenile-onset HPP patients treated with Asfotase alfa demonstrated decreases in osteoid volume and thickness indicating improved bone mineralization.
Mechanism of ActionHPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
ToxicityThere are no available human data on Asfotase Alfa use in pregnant women to inform a drug associated risk. In animal reproduction studies, Asfotase Alfa administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no evidence of fetotoxicity, embryolethality or teratogenicity at doses causing plasma exposures up to 21 and 24 times, respectively, the exposure at the recommended human dose.
MetabolismNA
AbsorptionNA
Volume of DistributionNA
ClearanceNA
CategoriesEnzymes Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetSphingosine 1-phosphate receptor 1; Pyrophosphate
Information of corresponding available drug in the market
Brand NameStrensiq
CompanyAlexion Pharma Ghbh
Brand DiscriptionNA
Prescribed forSTRENSIQâ„¢ is indicated for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP).
Chemical NameNA
Formulation40 mg
Physcial AppearanceSolution
Route of AdministrationSubcutaneous
Recommended DosageThe recommended dosage regimen of STRENSIQ for the treatment of perinatal/infantileonset HPP is 6 mg/kg per week administered subcutaneously as either: 2 mg/kg three times per week, or 1 mg/kg six times per week. Injection site reactions may limit the tolerability of the six times per week regimen
ContraindicationNA
Side EffectsHypersensitivity Reactions; Lipodystrophy; Ectopic Calcifications.
Useful Linkhttp://www.rxlist.com/strensiq-drug/side-effects-interactions.htm
PubMed ID26893260, 26529632, 22397657
3-D StructureTh1166 (View) or (Download)
Primary information
ID1699
ThPP IDTh1166
Therapeutic Peptide/Protein NameAsfotase Alfa
SequenceLVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTV view full sequnce in fasta
Functional ClassificationIa
Molecular Weight180006
Chemical FormulaC7108H11008N1968O2206S62
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half LifeApproximately 5 days.
DescriptionAsfotase Alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body’s ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015.
Indication/DiseaseIndicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
PharmacodynamicsPerinatal/infantile- and juvenile-onset HPP patients treated with Asfotase alfa had reductions in plasma TNSALP (tissue non-specific alkaline phosphatase) substrates, PPi and pyridoxal 5'-phosphate (PLP) within 6 to 12 weeks of treatment. Reductions in plasma PPi and PLP levels did not correlate with clinical outcomes. Bone biopsy data from perinatal/infantile-onset and juvenile-onset HPP patients treated with Asfotase alfa demonstrated decreases in osteoid volume and thickness indicating improved bone mineralization.
Mechanism of ActionHPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
ToxicityThere are no available human data on Asfotase Alfa use in pregnant women to inform a drug associated risk. In animal reproduction studies, Asfotase Alfa administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no evidence of fetotoxicity, embryolethality or teratogenicity at doses causing plasma exposures up to 21 and 24 times, respectively, the exposure at the recommended human dose.
MetabolismNA
AbsorptionNA
Volume of DistributionNA
ClearanceNA
CategoriesEnzymes Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetSphingosine 1-phosphate receptor 1; Pyrophosphate
Information of corresponding available drug in the market
Brand NameStrensiq
CompanyAlexion Pharma Ghbh
Brand DiscriptionNA
Prescribed forSTRENSIQâ„¢ is indicated for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP).
Chemical NameNA
Formulation40 mg
Physcial AppearanceSolution
Route of AdministrationSubcutaneous
Recommended DosageThe recommended dosage regimen of STRENSIQ for the treatment of perinatal/infantileonset HPP is 6 mg/kg per week administered subcutaneously as either: 2 mg/kg three times per week, or 1 mg/kg six times per week. Injection site reactions may limit the tolerability of the six times per week regimen
ContraindicationNA
Side EffectsHypersensitivity Reactions; Lipodystrophy; Ectopic Calcifications.
Useful Linkhttp://www.rxlist.com/strensiq-drug/side-effects-interactions.htm
PubMed ID26893260, 26529632, 22397658
3-D StructureTh1166 (View) or (Download)
Primary information
ID1700
ThPP IDTh1166
Therapeutic Peptide/Protein NameAsfotase Alfa
SequenceLVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTV view full sequnce in fasta
Functional ClassificationIa
Molecular Weight180007
Chemical FormulaC7108H11008N1968O2206S63
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half LifeApproximately 5 days.
DescriptionAsfotase Alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body’s ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015.
Indication/DiseaseIndicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
PharmacodynamicsPerinatal/infantile- and juvenile-onset HPP patients treated with Asfotase alfa had reductions in plasma TNSALP (tissue non-specific alkaline phosphatase) substrates, PPi and pyridoxal 5'-phosphate (PLP) within 6 to 12 weeks of treatment. Reductions in plasma PPi and PLP levels did not correlate with clinical outcomes. Bone biopsy data from perinatal/infantile-onset and juvenile-onset HPP patients treated with Asfotase alfa demonstrated decreases in osteoid volume and thickness indicating improved bone mineralization.
Mechanism of ActionHPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
ToxicityThere are no available human data on Asfotase Alfa use in pregnant women to inform a drug associated risk. In animal reproduction studies, Asfotase Alfa administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no evidence of fetotoxicity, embryolethality or teratogenicity at doses causing plasma exposures up to 21 and 24 times, respectively, the exposure at the recommended human dose.
MetabolismNA
AbsorptionNA
Volume of DistributionNA
ClearanceNA
CategoriesEnzymes Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetSphingosine 1-phosphate receptor 1; Pyrophosphate
Information of corresponding available drug in the market
Brand NameStrensiq
CompanyAlexion Pharmaceuticals Inc.
Brand DiscriptionNA
Prescribed forSTRENSIQâ„¢ is indicated for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP).
Chemical NameNA
Formulation80 mg/.8mL
Physcial AppearanceSolution
Route of AdministrationSubcutaneous
Recommended DosageThe recommended dosage regimen of STRENSIQ for the treatment of perinatal/infantileonset HPP is 6 mg/kg per week administered subcutaneously as either: 2 mg/kg three times per week, or 1 mg/kg six times per week. Injection site reactions may limit the tolerability of the six times per week regimen
ContraindicationNA
Side EffectsHypersensitivity Reactions; Lipodystrophy; Ectopic Calcifications.
Useful Linkhttp://www.rxlist.com/strensiq-drug/side-effects-interactions.htm
PubMed ID26893260, 26529632, 22397659
3-D StructureTh1166 (View) or (Download)
Primary information
ID1701
ThPP IDTh1166
Therapeutic Peptide/Protein NameAsfotase Alfa
SequenceLVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTV view full sequnce in fasta
Functional ClassificationIa
Molecular Weight180008
Chemical FormulaC7108H11008N1968O2206S64
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half LifeApproximately 5 days.
DescriptionAsfotase Alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body’s ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015.
Indication/DiseaseIndicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
PharmacodynamicsPerinatal/infantile- and juvenile-onset HPP patients treated with Asfotase alfa had reductions in plasma TNSALP (tissue non-specific alkaline phosphatase) substrates, PPi and pyridoxal 5'-phosphate (PLP) within 6 to 12 weeks of treatment. Reductions in plasma PPi and PLP levels did not correlate with clinical outcomes. Bone biopsy data from perinatal/infantile-onset and juvenile-onset HPP patients treated with Asfotase alfa demonstrated decreases in osteoid volume and thickness indicating improved bone mineralization.
Mechanism of ActionHPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
ToxicityThere are no available human data on Asfotase Alfa use in pregnant women to inform a drug associated risk. In animal reproduction studies, Asfotase Alfa administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no evidence of fetotoxicity, embryolethality or teratogenicity at doses causing plasma exposures up to 21 and 24 times, respectively, the exposure at the recommended human dose.
MetabolismNA
AbsorptionNA
Volume of DistributionNA
ClearanceNA
CategoriesEnzymes Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetSphingosine 1-phosphate receptor 1; Pyrophosphate
Information of corresponding available drug in the market
Brand NameStrensiq
CompanyAlexion Pharmaceuticals Inc.
Brand DiscriptionNA
Prescribed forSTRENSIQâ„¢ is indicated for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP).
Chemical NameNA
Formulation40 mg/mL
Physcial AppearanceSolution
Route of AdministrationSubcutaneous
Recommended DosageThe recommended dosage regimen of STRENSIQ for the treatment of perinatal/infantileonset HPP is 6 mg/kg per week administered subcutaneously as either: 2 mg/kg three times per week, or 1 mg/kg six times per week. Injection site reactions may limit the tolerability of the six times per week regimen
ContraindicationNA
Side EffectsHypersensitivity Reactions; Lipodystrophy; Ectopic Calcifications.
Useful Linkhttp://www.rxlist.com/strensiq-drug/side-effects-interactions.htm
PubMed ID26893260, 26529632, 22397660
3-D StructureTh1166 (View) or (Download)
Primary information
ID1702
ThPP IDTh1166
Therapeutic Peptide/Protein NameAsfotase Alfa
SequenceLVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTV view full sequnce in fasta
Functional ClassificationIa
Molecular Weight180009
Chemical FormulaC7108H11008N1968O2206S65
Isoelectric PointNA
HydrophobicityNA
Melting Point (℃)NA
Half LifeApproximately 5 days.
DescriptionAsfotase Alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body’s ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015.
Indication/DiseaseIndicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).
PharmacodynamicsPerinatal/infantile- and juvenile-onset HPP patients treated with Asfotase alfa had reductions in plasma TNSALP (tissue non-specific alkaline phosphatase) substrates, PPi and pyridoxal 5'-phosphate (PLP) within 6 to 12 weeks of treatment. Reductions in plasma PPi and PLP levels did not correlate with clinical outcomes. Bone biopsy data from perinatal/infantile-onset and juvenile-onset HPP patients treated with Asfotase alfa demonstrated decreases in osteoid volume and thickness indicating improved bone mineralization.
Mechanism of ActionHPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
ToxicityThere are no available human data on Asfotase Alfa use in pregnant women to inform a drug associated risk. In animal reproduction studies, Asfotase Alfa administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no evidence of fetotoxicity, embryolethality or teratogenicity at doses causing plasma exposures up to 21 and 24 times, respectively, the exposure at the recommended human dose.
MetabolismNA
AbsorptionNA
Volume of DistributionNA
ClearanceNA
CategoriesEnzymes Alimentary Tract and Metabolism
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNA
TargetSphingosine 1-phosphate receptor 1; Pyrophosphate
Information of corresponding available drug in the market
Brand NameStrensiq
CompanyAlexion Pharmaceuticals Inc.
Brand DiscriptionNA
Prescribed forSTRENSIQâ„¢ is indicated for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP).
Chemical NameNA
Formulation28 mg/.7mL
Physcial AppearanceSolution
Route of AdministrationSubcutaneous
Recommended DosageThe recommended dosage regimen of STRENSIQ for the treatment of perinatal/infantileonset HPP is 6 mg/kg per week administered subcutaneously as either: 2 mg/kg three times per week, or 1 mg/kg six times per week. Injection site reactions may limit the tolerability of the six times per week regimen
ContraindicationNA
Side EffectsHypersensitivity Reactions; Lipodystrophy; Ectopic Calcifications.
Useful Linkhttp://www.rxlist.com/strensiq-drug/side-effects-interactions.htm
PubMed ID26893260, 26529632, 22397661
3-D StructureTh1166 (View) or (Download)