drug · enzyme

ELELYSO

updated 3mo ago

by $PFE PFIZER INC🔗 ChEMBL

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Enzyme drug with a maximum clinical stage of Approval (across all indications), with an approval for gaucher disease and metabolic disease and 1 investigational indication.

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Trials studying ELELYSO

phase4A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Diseasecompletedn=14

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brand

ELELYSO

trade names

Elelyso

generic

TALIGLUCERASE ALFA

MOA

12.1 Mechanism of Action Gaucher disease is an autosomal recessive disorder caused by mutations in the human glucocerebrosidase gene, which results in a reduced activity of the lysosomal enzyme glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency results in accumulation of substrate glucocerebroside primarily in the lysosomal compartment of macrophages, giving rise to foam cells or "Gaucher cells," which accumulate in the liver, spleen and bone marrow. ELELYSO, an enzyme replacement therapy, is a recombinant analog of human lysosomal glucocerebrosidase that catalyzes the hydrolysis of glucocerebroside to glucose and ceramide, reducing the amount of accumulated glucocerebroside. ELELYSO uptake into cellular lysosomes is mediated by binding of ELELYSO mannose oligosaccharide chains to specific mannose receptors on the cell surface leading to internalization and subsequent transport to the lysosomes.

indication

1 INDICATIONS AND USAGE ELELYSO is indicated for the treatment of patients 4 years of age and older with a confirmed diagnosis of Type 1 Gaucher disease. ELELYSO is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease ( 1 ).

Data sources

ClinicalTrials.gov · OpenTargets

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drug · enzyme

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ELELYSO

updated 3mo ago

by $PFE PFIZER INC🔗 ChEMBL

community read0 votes

🐶 often sniffed alongside

top 3

Enzyme drug with a maximum clinical stage of Approval (across all indications), with an approval for gaucher disease and metabolic disease and 1 investigational indication.

all catalysts(0)

nothing on the calendar yet

Trials studying ELELYSO

phase4A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Diseasecompletedn=14

News(0)

no news has mentioned this drug yet

brand

ELELYSO

trade names

Elelyso

generic

TALIGLUCERASE ALFA

MOA

indication

Data sources

ClinicalTrials.gov · OpenTargets

last refreshed 20m ago

Found something off?

comments(0)

5-min edit window · permanent after that

no comments yet — first sniff?

ELELYSO

🐶 often sniffed alongside

all catalysts(0)

Trials studying ELELYSO

News(0)

Report a data issue

comments(0)

ELELYSO

🐶 often sniffed alongside

all catalysts(0)

Trials studying ELELYSO

News(0)

Report a data issue

comments(0)