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Details

Stereochemistry ABSOLUTE
Molecular Formula C11H19NO9
Molecular Weight 309.2699
Optical Activity UNSPECIFIED
Defined Stereocenters 5 / 5
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of ACENEURAMIC ACID

SMILES

CC(=O)N[C@H]([C@@H](O)CC(=O)C(O)=O)[C@@H](O)[C@H](O)[C@H](O)CO

InChI

InChIKey=KBGAYAKRZNYFFG-BOHATCBPSA-N
InChI=1S/C11H19NO9/c1-4(14)12-8(5(15)2-6(16)11(20)21)10(19)9(18)7(17)3-13/h5,7-10,13,15,17-19H,2-3H2,1H3,(H,12,14)(H,20,21)/t5-,7+,8+,9+,10+/m0/s1

HIDE SMILES / InChI

Molecular Formula C11H19NO9
Molecular Weight 309.2699
Charge 0
Count
MOL RATIO 1 MOL RATIO (average)
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 5 / 5
E/Z Centers 0
Optical Activity UNSPECIFIED

Description

Aceneuramic acid (also known as N-Acetylneuraminic Acid, Neu5Ac, the UX 001 tablets) prolonged released had been filed a marketing authorization application with the European Medicines Agency by Ultragenyx in the EU for the treatment of hereditary inclusion body myopathy (HIBM), a rare, progressive muscle-wasting disease. This compound, a muscle protein stimulant, is also in phase II of the clinical trial for the treatment thrombocytopenia. In addition, recently was made studies, which had shown, that existed the significant positive correlation between serum and salivary sialic acid levels in oral squamous cell carcinoma (OSCC) patients.

Approval Year

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Unknown
Primary
Unknown
Diagnostic
Unknown

Cmax

ValueDoseCo-administeredAnalytePopulation
0.624 μg/mL
2000 mg single, oral
ACENEURAMIC ACID plasma
Homo sapiens
0.285 μg/mL
2000 mg 3 times / day multiple, oral
ACENEURAMIC ACID plasma
Homo sapiens

AUC

ValueDoseCo-administeredAnalytePopulation
2.279 μg × h/mL
2000 mg single, oral
ACENEURAMIC ACID plasma
Homo sapiens
1.731 μg × h/mL
2000 mg 3 times / day multiple, oral
ACENEURAMIC ACID plasma
Homo sapiens

T1/2

ValueDoseCo-administeredAnalytePopulation
7.089 h
2000 mg single, oral
ACENEURAMIC ACID plasma
Homo sapiens

Doses

AEs

Overview

CYP3A4CYP2C9CYP2D6hERG


OverviewOther

Other InhibitorOther SubstrateOther Inducer


Drug as perpetrator​

Drug as victim

Tox targets

PubMed

Sample Use Guides

In Vivo Use Guide
Subjects will take 4 tablets (500 mg aceneuramic acid extended-release (Ace-ER) each for 2 g per dose) orally 3 times per day (TID).The dose should be taken with food (i.e. within 30 minutes after a meal or snack).
Route of Administration: Oral
In Vitro Use Guide
Unknown
Substance Class Chemical
Record UNII
GZP2782OP0
Record Status Validated (UNII)
Record Version