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Development Status

US Approved Rx

3

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Primary Target

Ceramide glucosyltransferase

1

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Highest Phase

Approved

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Approval Year

2010

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2012

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2014

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Condition

Gaucher's disease type I

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Treatment Modality

Secondary

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CNS Activity

CNS Non-penetrant

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Originator

Genzyme

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Substance Class

Protein

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Chemical

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Code System

CAS

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ChEMBL

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DRUG BANK

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EMA ASSESSMENT REPORTS

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EU-Orphan Drug

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ATC Level 1

ALIMENTARY TRACT AND METABOLISM

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ATC Level 2

OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS

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ATC Level 3

OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS

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ATC Level 4

Enzymes

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Substance Form

Principal Form

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Salts, Hydrates, Esters, etc.

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Search results for "EU ORPHAN DRUG|Positive|Treatment of Gaucher disease" in comments (approximate match)

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TALIGLUCERASE ALFA

0R4NLX88O4

Subunit 1 (506) Similarity Search

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Subunit 1 (506) Similarity Search

Status:

US Approved Rx (2012)

First approved in 2012

Class:

PROTEIN

VELAGLUCERASE ALFA

23HYE36B0I

Subunit 1 (497) Similarity Search

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Subunit 1 (497) Similarity Search

Status:

US Approved Rx (2010)

First approved in 2010

Class:

PROTEIN

ELIGLUSTAT TARTRATE

N0493335P3

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Status:

US Approved Rx (2014)

First approved in 2014

Class (Stereo):

CHEMICAL (ABSOLUTE)

Targets:

Ceramide glucosyltransferase

Conditions:

Gaucher's disease type I

Eliglustat, marketed by Genzyme as CERDELGA, is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease who are CYP2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers (PMs) as dete...

cted by an FDA-cleared test.

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