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Chemistry

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Also known as: Eliglustat hemitartrate, Cerdelga, 928659-70-5, Agalsidase beta, Eliglustat (hemitartrate), Genz-112638
Molecular Formula
C50H78N4O14
Molecular Weight
959.2  g/mol
InChI Key
KUBARPMUNHKBIQ-VTHUDJRQSA-N
FDA UNII
N0493335P3

Eliglustat
Agalsidase beta is a recombinant human -galactosidase A similar to [agalsidase alfa]. While patients generally do not experience a clinically significant difference in outcomes between the two drugs, some patients may experience greater benefit with agalsidase beta. Use of agalsidase beta has decreased in Europe, in favor of agalsidase alfa, after a contamination event in 2009. Agalsidase beta was granted FDA approval on 24 April 2003.
1 2D Structure

Eliglustat

2 Identification
2.1 Computed Descriptors
2.1.1 IUPAC Name
N-[(1R,2R)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-pyrrolidin-1-ylpropan-2-yl]octanamide;(2R,3R)-2,3-dihydroxybutanedioic acid
2.1.2 InChI
InChI=1S/2C23H36N2O4.C4H6O6/c2*1-2-3-4-5-6-9-22(26)24-19(17-25-12-7-8-13-25)23(27)18-10-11-20-21(16-18)29-15-14-28-20;5-1(3(7)8)2(6)4(9)10/h2*10-11,16,19,23,27H,2-9,12-15,17H2,1H3,(H,24,26);1-2,5-6H,(H,7,8)(H,9,10)/t2*19-,23-;1-,2-/m111/s1
2.1.3 InChI Key
KUBARPMUNHKBIQ-VTHUDJRQSA-N
2.1.4 Canonical SMILES
CCCCCCCC(=O)NC(CN1CCCC1)C(C2=CC3=C(C=C2)OCCO3)O.CCCCCCCC(=O)NC(CN1CCCC1)C(C2=CC3=C(C=C2)OCCO3)O.C(C(C(=O)O)O)(C(=O)O)O
2.1.5 Isomeric SMILES
CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@@H](C2=CC3=C(C=C2)OCCO3)O.CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@@H](C2=CC3=C(C=C2)OCCO3)O.[C@@H]([C@H](C(=O)O)O)(C(=O)O)O
2.2 Other Identifiers
2.2.1 UNII
N0493335P3
2.3 Synonyms
2.3.1 MeSH Synonyms

1. Cerdelga

2. Eliglustat

3. Genz-112638

2.3.2 Depositor-Supplied Synonyms

1. Eliglustat Hemitartrate

2. Cerdelga

3. 928659-70-5

4. Agalsidase Beta

5. Eliglustat (hemitartrate)

6. Genz-112638

7. 104138-64-9

8. Eliglustat Tartrate [usan]

9. Eliglustat (tartrate)

10. Genz 112638

11. 928659-70-5 (tartrate)

12. N0493335p3

13. N-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-pyrrolidin-1-ylpropan-2-yl]octanamide;(2r,3r)-2,3-dihydroxybutanedioic Acid

14. N-((1r,2r)-1-(2,3-dihydrobenzo[b][1,4]dioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl)octanamide Hemi((2r,3r)-2,3-dihydroxysuccinate)

15. Unii-n0493335p3

16. Cerdelga (tn)

17. Eliglustat L-tartrate

18. Eliglustat Tartrate [mi]

19. Chebi:83353

20. Eliglustat Tartrate (jan/usan)

21. Dtxsid50239166

22. Eliglustat Tartrate [jan]

23. Eliglustat Tartrategenz-112638

24. Eliglustat Tartrate [who-dd]

25. Hy-14885a

26. Genz-112638;eliglustat Hemitartrate

27. Cs-5423

28. Ex-a2301-1

29. Eliglustat Hemitartrate (genz-112638)

30. Eliglustat Tartrate [orange Book]

31. Ac-35333

32. Bis(n-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(pyrrolidin-1- Ylmethyl)ethyl)octanamide) (2r,3r)-2,3-dihydroxybutanedioate

33. Octanamide, N-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1- Pyrrolidinylmethyl)ethyl)-, (2r,3r)-2,3-dihydroxybutanedioate (2:1)

34. Cerdelga Component Eliglustat Tartrate

35. S4433

36. C16736

37. D09894

38. Eliglustat Tartrate Component Of Cerdelga

39. Q27156779

40. (1r,2r)-octanoic Acid(2-(2',3'-dihydro-benzo(1,4) Dioxin-6'-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl)-amide-l-tartaric Acid Salt

41. Bis{1-[(2r,3r)-3-(2,3-dihydro-1,4-benzodioxin-6-yl)-3-hydroxy-2-(octanoylamino)propyl]pyrrolidinium} (2r,3r)-2,3-dihydroxysuccinate

42. Bis{n-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl]octanamide} (2r,3r)-2,3-dihydroxysuccinic Acid

43. N-((1r,2r)-1-(2,3-dihydrobenzo[b][1,4]dioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl)octanamide ((2r,3r)-2,3-dihydroxysuccinate)(2:1)

44. Octanamide, N-((1r,2r)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1-pyrrolidinylmethyl)ethyl)-, (2r,3r)-2,3-dihydroxybutanedioate (2:1)

2.4 Create Date
2011-06-02
3 Chemical and Physical Properties
Molecular Weight 959.2 g/mol
Molecular Formula C50H78N4O14
Hydrogen Bond Donor Count8
Hydrogen Bond Acceptor Count16
Rotatable Bond Count25
Exact Mass958.55145317 g/mol
Monoisotopic Mass958.55145317 g/mol
Topological Polar Surface Area257 Ų
Heavy Atom Count68
Formal Charge0
Complexity617
Isotope Atom Count0
Defined Atom Stereocenter Count6
Undefined Atom Stereocenter Count0
Defined Bond Stereocenter Count0
Undefined Bond Stereocenter Count0
Covalently Bonded Unit Count3
4 Drug and Medication Information
4.1 Drug Indication

Agalsidase beta is indicated in the treatment of Fabry disease.


FDA Label


Cerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs).


Treatment of Gaucher disease Type 1 and Type 3, Treatment of Gaucher disease Type 2


Fabrazyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (-galactosidase-A deficiency).


5 Pharmacology and Biochemistry
5.1 Pharmacology

Agalsidase beta is a recombinant human -galactosidase A used as enzyme replacement therapy in the treatment of Fabry disease. It has a long duration of action and a wide therapeutic index. Patients should be counselled regarding the risk of infusion related reactions and hypersensitivity.


5.2 MeSH Pharmacological Classification

Enzyme Inhibitors

Compounds or agents that combine with an enzyme in such a manner as to prevent the normal substrate-enzyme combination and the catalytic reaction. (See all compounds classified as Enzyme Inhibitors.)


5.3 FDA Pharmacological Classification
5.3.1 Pharmacological Classes
alpha-Glucosidases [CS]; Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme [EPC]
5.4 ATC Code

A16AX10


A16AB04


A - Alimentary tract and metabolism

A16 - Other alimentary tract and metabolism products

A16A - Other alimentary tract and metabolism products

A16AB - Enzymes

A16AB04 - Agalsidase beta


5.5 Absorption, Distribution and Excretion

Absorption

A 1 mg/kg dose of agalsidase beta with a mean infusion length of 115 minutes reaches a Cmax 5.0 1.1 g/mL with an AUC of 496 137 g\*min/mL.


Route of Elimination

After nonspecific proteolysis, the amino acids from protein drugs are reused for protein synthesis or further broken down and eliminated by the kidneys.


Volume of Distribution

A 1 mg/kg dose of agalsidase beta with a mean infusion length of 115 minutes has a VSS of 112 13 mL/kg.


Clearance

A 1 mg/kg dose of agalsidase beta with a mean infusion length of 115 minutes has a clearance of 2.1 0.7 mL/min/kg.


5.6 Metabolism/Metabolites

Data regarding the metabolism of agalsidase beta is not readily available. However, protein drugs are expected to be degraded by proteases and other catalytic enzymes to smaller peptides and amino acids.


5.7 Biological Half-Life

agalsidase beta has a half like of 67 12 min for a 1 mg/kg dose with a mean infusion length of 115 minutes.


5.8 Mechanism of Action

-galactosidase A is uptaken by cells via the mannose 6 phosphate receptor. Agalsidase beta hydrolyzes globotriaosylceramide and other glycosphingolipids that would normally be hydrolyzed by endogenous -galactosidase A. Preventing the accumulation of glycosphingolipids prevents or reduces the severity of manifestations of Fabry disease such as renal failure, cardiomyopathy, or cerebrovascular events.


API Reference Price

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21-Jun-2021
20-Mar-2023
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ABOUT THIS PAGE

Looking for 928659-70-5 / Eliglustat API manufacturers, exporters & distributors?

Eliglustat manufacturers, exporters & distributors 1

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PharmaCompass offers a list of Eliglustat API manufacturers, exporters & distributors, which can be sorted by GMP, USDMF, JDMF, KDMF, CEP (COS), WC, Price,and more, enabling you to easily find the right Eliglustat manufacturer or Eliglustat supplier for your needs.

Send us enquiries for free, and we will assist you in establishing a direct connection with your preferred Eliglustat manufacturer or Eliglustat supplier.

PharmaCompass also assists you with knowing the Eliglustat API Price utilized in the formulation of products. Eliglustat API Price is not always fixed or binding as the Eliglustat Price is obtained through a variety of data sources. The Eliglustat Price can also vary due to multiple factors, including market conditions, regulatory modifications, or negotiated pricing deals.

API | Excipient name

Eliglustat

Synonyms

Eliglustat hemitartrate, Cerdelga, 928659-70-5, Agalsidase beta, Eliglustat (hemitartrate), Genz-112638

Cas Number

928659-70-5

Unique Ingredient Identifier (UNII)

N0493335P3

About Eliglustat

Agalsidase beta is a recombinant human -galactosidase A similar to [agalsidase alfa]. While patients generally do not experience a clinically significant difference in outcomes between the two drugs, some patients may experience greater benefit with agalsidase beta. Use of agalsidase beta has decreased in Europe, in favor of agalsidase alfa, after a contamination event in 2009. Agalsidase beta was granted FDA approval on 24 April 2003.

Eliglustat Manufacturers

A Eliglustat manufacturer is defined as any person or entity involved in the manufacture, preparation, processing, compounding or propagation of Eliglustat, including repackagers and relabelers. The FDA regulates Eliglustat manufacturers to ensure that their products comply with relevant laws and regulations and are safe and effective to use. Eliglustat API Manufacturers are required to adhere to Good Manufacturing Practices (GMP) to ensure that their products are consistently manufactured to meet established quality criteria.

click here to find a list of Eliglustat manufacturers with USDMF, JDMF, KDMF, CEP, GMP, COA and API Price related information on PhamaCompass.

Eliglustat Suppliers

A Eliglustat supplier is an individual or a company that provides Eliglustat active pharmaceutical ingredient (API) or Eliglustat finished formulations upon request. The Eliglustat suppliers may include Eliglustat API manufacturers, exporters, distributors and traders.

click here to find a list of Eliglustat suppliers with USDMF, JDMF, KDMF, CEP, GMP, COA and API Price related information on PharmaCompass.

Eliglustat USDMF

A Eliglustat DMF (Drug Master File) is a document detailing the whole manufacturing process of Eliglustat active pharmaceutical ingredient (API) in detail. Different forms of Eliglustat DMFs exist exist since differing nations have different regulations, such as Eliglustat USDMF, ASMF (EDMF), JDMF, CDMF, etc.

A Eliglustat DMF submitted to regulatory agencies in the US is known as a USDMF. Eliglustat USDMF includes data on Eliglustat's chemical properties, information on the facilities and procedures used, and details about packaging and storage. The Eliglustat USDMF is kept confidential to protect the manufacturer’s intellectual property.

click here to find a list of Eliglustat suppliers with USDMF on PharmaCompass.

Eliglustat KDMF

In Korea, the Ministry of Food and Drug Safety (MFDS) is in charge of regulating pharmaceutical products and services.

Pharmaceutical companies submit a Eliglustat Drug Master File in Korea (Eliglustat KDMF) to the MFDS, which includes comprehensive information about the production, processing, facilities, materials, packaging, and testing of Eliglustat. The MFDS reviews the Eliglustat KDMF as part of the drug registration process and uses the information provided in the Eliglustat KDMF to evaluate the safety and efficacy of the drug.

After submitting a Eliglustat KDMF to the MFDS, the registered manufacturer can provide importers or distributors with the registration number without revealing confidential information to Korean business partners. Applicants seeking to register their Eliglustat API can apply through the Korea Drug Master File (KDMF).

click here to find a list of Eliglustat suppliers with KDMF on PharmaCompass.

Eliglustat WC

A Eliglustat written confirmation (Eliglustat WC) is an official document issued by a regulatory agency to a Eliglustat manufacturer, verifying that the manufacturing facility of a Eliglustat active pharmaceutical ingredient (API) adheres to the Good Manufacturing Practices (GMP) regulations of the importing country. When exporting Eliglustat APIs or Eliglustat finished pharmaceutical products to another nation, regulatory agencies frequently require a Eliglustat WC (written confirmation) as part of the regulatory process.

click here to find a list of Eliglustat suppliers with Written Confirmation (WC) on PharmaCompass.

Eliglustat NDC

National Drug Code is a comprehensive database maintained by the FDA that contains information on all drugs marketed in the US. This directory includes information about finished drug products, unfinished drug products, and compounded drug products, including those containing Eliglustat as an active pharmaceutical ingredient (API).

Finished drug products

The FDA updates the NDC directory daily. The NDC numbers for Eliglustat API and other APIs are published in this directory by the FDA.

Unfinished drugs

The NDC unfinished drugs database includes product listing information submitted for all unfinished drugs, such as active pharmaceutical ingredients (APIs), drugs intended for further processing and bulk drug substances for compounding.

Pharmaceutical companies that manufacture Eliglustat as an active pharmaceutical ingredient (API) must furnish the FDA with an updated record of all drugs that they produce, prepare, propagate, compound, or process for commercial distribution in the US at their facilities.

Compounded drug products

The NDC directory also contains data on finished compounded human drug products that contain Eliglustat and are produced by outsourcing facilities. While these outsourcing facilities are not mandated to assign a Eliglustat NDC to their finished compounded human drug products, they may choose to do so.

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Eliglustat GMP

Eliglustat Active pharmaceutical ingredient (API) is produced in GMP-certified manufacturing facility.

GMP stands for Good Manufacturing Practices, which is a system used in the pharmaceutical industry to make sure that goods are regularly produced and monitored in accordance with quality standards. The FDA’s current Good Manufacturing Practices requirements are referred to as cGMP or current GMP which indicates that the company follows the most recent GMP specifications. The World Health Organization (WHO) has its own set of GMP guidelines, called the WHO GMP. Different countries can also set their own guidelines for GMP like China (Chinese GMP) or the EU (EU GMP).

PharmaCompass offers a list of Eliglustat GMP manufacturers, exporters & distributors, which can be sorted by USDMF, JDMF, KDMF, CEP (COS), WC, API price, and more, enabling you to easily find the right Eliglustat GMP manufacturer or Eliglustat GMP API supplier for your needs.

Eliglustat CoA

A Eliglustat CoA (Certificate of Analysis) is a formal document that attests to Eliglustat's compliance with Eliglustat specifications and serves as a tool for batch-level quality control.

Eliglustat CoA mostly includes findings from lab analyses of a specific batch. For each Eliglustat CoA document that a company creates, the USFDA specifies specific requirements, such as supplier information, material identification, transportation data, evidence of conformity and signature data.

Eliglustat may be tested according to a variety of international standards, such as European Pharmacopoeia (Eliglustat EP), Eliglustat JP (Japanese Pharmacopeia) and the US Pharmacopoeia (Eliglustat USP).

Inform the supplier about your product requirements, specifying if you need a product with particular monograph like EP (Ph. Eur.), USP, JP, BP, or any other quality. In addition, clarify whether you need hydrochloride (HCl), anhydricum, base, micronisatum or a specific level of purity. To find reputable suppliers, utilize the filters and select those certified by GMP, FDA, or any other certification as per your requirement.
For your convenience, we have listed synonyms and CAS numbers to help you find the best supplier. The use of synonyms and CAS numbers can be helpful in identifying potential suppliers, but it is crucial to note that they might not always indicate the exact same product. It is important to confirm the product details with the supplier before making a purchase to ensure that it meets your requirements.
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