ACENEURAMIC ACID

First approved in 2022

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 Search

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

Stereochemistry	ABSOLUTE
Additional Stereochemistry	No
Defined Stereocenters	5 / 5
E/Z Centers	0
Optical Activity	UNSPECIFIED

Description
Sources: https://www.ncbi.nlm.nih.gov/pubmed/27854209 | https://www.epgonline.org/global/news/ux-001--aceneuramic-acid-prolonged-release--filed-with-eu-for-hereditary-inclusion-body-myopathy--ultragenyx-pharmaceutical.html | https://www.ncbi.nlm.nih.gov/pubmed/28928781

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

Condition	Modality	Highest Phase	Product
GNE myopathy 4 Sources: https://www.ncbi.nlm.nih.gov/pubmed/27854209	Primary	Phase III 665	Unknown Approved Use Unknown
Thrombocytopenia 2 Sources: https://clinicaltrials.gov/ct2/show/NCT02845609	Primary	Phase II 1147	Unknown Approved Use Unknown
Oral squamous cell carcinoma 6 Sources: https://www.ncbi.nlm.nih.gov/pubmed/28928781	Diagnostic	Natural Metabolite	Unknown Approved Use Unknown

C_max

Value	Dose	Co-administered	Analyte	Population
0.624 μg/mL EXPERIMENT https://transmedcomms.biomedcentral.com/articles/10.1186/s41231-018-0025-0	2000 mg single, oral dose: 2000 mg route of administration: Oral experiment type: SINGLE co-administered:		ACENEURAMIC ACID plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: UNKNOWN food status: FASTED
0.285 μg/mL EXPERIMENT https://transmedcomms.biomedcentral.com/articles/10.1186/s41231-018-0025-0	2000 mg 3 times / day multiple, oral dose: 2000 mg route of administration: Oral experiment type: MULTIPLE co-administered:		ACENEURAMIC ACID plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: UNKNOWN food status: UNKNOWN

AUC

Value	Dose	Co-administered	Analyte	Population
2.279 μg × h/mL EXPERIMENT https://transmedcomms.biomedcentral.com/articles/10.1186/s41231-018-0025-0	2000 mg single, oral dose: 2000 mg route of administration: Oral experiment type: SINGLE co-administered:		ACENEURAMIC ACID plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: UNKNOWN food status: FASTED
1.731 μg × h/mL EXPERIMENT https://transmedcomms.biomedcentral.com/articles/10.1186/s41231-018-0025-0	2000 mg 3 times / day multiple, oral dose: 2000 mg route of administration: Oral experiment type: MULTIPLE co-administered:		ACENEURAMIC ACID plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: UNKNOWN food status: UNKNOWN

T_1/2

Value	Dose	Co-administered	Analyte	Population
7.089 h EXPERIMENT https://transmedcomms.biomedcentral.com/articles/10.1186/s41231-018-0025-0	2000 mg single, oral dose: 2000 mg route of administration: Oral experiment type: SINGLE co-administered:		ACENEURAMIC ACID plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: UNKNOWN food status: FASTED

Doses

Dose	Population	Adverse events
6 g 1 times / day steady, oral Highest studied dose Dose: 6 g, 1 times / day Route: oral Route: steady Dose: 6 g, 1 times / day Sources: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5271423/	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M+F Food Status: UNKNOWN Sources: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5271423/	Sources: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5271423/
2 g 3 times / day steady, oral Studied dose Dose: 2 g, 3 times / day Route: oral Route: steady Dose: 2 g, 3 times / day Sources: https://clinicaltrials.gov/ct2/show/NCT02377921?term=ACENEURAMIC+ACID&rslt=With&draw=2&rank=3	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M+F Food Status: UNKNOWN Sources: https://clinicaltrials.gov/ct2/show/NCT02377921?term=ACENEURAMIC+ACID&rslt=With&draw=2&rank=3	Other AEs: Acute myocardial infarction, Gastritis... Other AEs: Acute myocardial infarction (serious, 2.2%) Gastritis (serious, 2.2%) Sources: https://clinicaltrials.gov/ct2/show/NCT02377921?term=ACENEURAMIC+ACID&rslt=With&draw=2&rank=3

AEs

AE	Significance	Dose	Population
Acute myocardial infarction	serious, 2.2%	2 g 3 times / day steady, oral Studied dose Dose: 2 g, 3 times / day Route: oral Route: steady Dose: 2 g, 3 times / day Sources: https://clinicaltrials.gov/ct2/show/NCT02377921?term=ACENEURAMIC+ACID&rslt=With&draw=2&rank=3	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M+F Food Status: UNKNOWN Sources: https://clinicaltrials.gov/ct2/show/NCT02377921?term=ACENEURAMIC+ACID&rslt=With&draw=2&rank=3
Gastritis	serious, 2.2%	2 g 3 times / day steady, oral Studied dose Dose: 2 g, 3 times / day Route: oral Route: steady Dose: 2 g, 3 times / day Sources: https://clinicaltrials.gov/ct2/show/NCT02377921?term=ACENEURAMIC+ACID&rslt=With&draw=2&rank=3	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M+F Food Status: UNKNOWN Sources: https://clinicaltrials.gov/ct2/show/NCT02377921?term=ACENEURAMIC+ACID&rslt=With&draw=2&rank=3

Overview

CYP3A4	CYP2C9	CYP2D6	hERG
inducer 1087			inhibitor 2217

OverviewOther

Other Inhibitor	Other Substrate	Other Inducer
CYP19A1 429	P-gp 2052

Drug as perpetrator

Target	Modality	Activity	Metabolite	Clinical evidence
CYP19A1 430	inhibitor 4027 Sources: https://pubchem.ncbi.nlm.nih.gov/compound/14017587#section=Biological-Test-Results Page: 14.0	no
CYP3A4 2633	inducer 1966 Sources: https://pubchem.ncbi.nlm.nih.gov/compound/14017587#section=Biological-Test-Results Page: 17.0	no

Drug as victim

Target	Modality	Activity	Metabolite	Clinical evidence
P-gp 2052	substrate 4014 Sources: https://pubchem.ncbi.nlm.nih.gov/compound/14017587#section=Biological-Test-Results Page: 15 \| 16	no

Tox targets

Target	Modality	Activity	Metabolite	Clinical evidence
hERG 2263	inhibitor 2237 Sources: https://pubchem.ncbi.nlm.nih.gov/compound/14017587#section=Biological-Test-Results Page: 1.0

PubMed

Title	Date	PubMed
Identification and adipocyte differentiation-dependent expression of the unique disialic acid residue in an adipose tissue-specific glycoprotein, adipo Q.	2001-08-03	11382781
Differential contribution of sialic acid to the function of repolarizing K(+) currents in ventricular myocytes.	2001-08	11443045
A rapid, semi-automated method for detection of Galbeta1-4GlcNAc alpha2,6-sialyltransferase (EC 2.4.99.1) activity using the lectin Sambucus nigra agglutinin.	2001-07	11447135
Redirection of sialic acid metabolism in genetically engineered Escherichia coli.	2001-07	11447132
Characterization and mutagenesis of the recombinant N-acetylneuraminate lyase from Clostridium perfringens: insights into the reaction mechanism.	2001-07	11432751
Identification and characterization of S2V, a novel putative siglec that contains two V set Ig-like domains and recruits protein-tyrosine phosphatases SHPs.	2001-06-29	11328818
De novo synthesis of a methylene-bridged Neu5Ac-alpha-(2,3)-gal C-disaccharide.	2001-06-15	11397161
Overexpression of lysosomal-type sialidase leads to suppression of metastasis associated with reversion of malignant phenotype in murine B16 melanoma cells.	2001-06-15	11351298
Genomics. Are there bugs in our genome?	2001-06-08	11358998
Capillary electrophoresis of sialic acid-containing glycoprotein. Effect of the heterogeneity of carbohydrate chains on glycoform separation using an alpha1-acid glycoprotein as a model.	2001-06-01	11403311
Role of galactosylation in the renal pathogenicity of murine immunoglobulin G3 monoclonal cryoglobulins.	2001-06-01	11369648
N-acetylneuraminic acid (NANA) stimulates in situ cyclic AMP production in tentacles of sea anemone (Aiptasia pallida): possible role in chemosensitization of nematocyst discharge.	2001-06	11441042
Siglecs in the immune system.	2001-06	11412300
Effect of papaya seed extract on microenvironment of cauda epididymis.	2001-06	11404801
A longitudinal study of salivary sialic acid in preterm infants: Comparison of human milk-fed versus formula-fed infants.	2001-06	11391340
Metabolic selection of glycosylation defects in human cells.	2001-06	11385460
Dominant inheritance of sialuria, an inborn error of feedback inhibition.	2001-06	11326336
Neuraminidase from a bacterial source enhances both HIV-1-mediated syncytium formation and the virus binding/entry process.	2001-05-25	11352665
Synthesis of C-3 nitrogen-containing derivatives of N-acetyl-alpha,beta-D-mannosamine as substrates for N-acetylneuraminic acid aldolase.	2001-05-18	11434370
Effects of sialic acid residues of transferrin on the binding with aluminum and iron studied by HPLC/high-resolution ICP-MS.	2001-05-03	11325539
Alcohol biomarkers: clinical significance and biochemical basis.	2001-05	11391052
Prenatal detection of free sialic acid storage disease: genetic and biochemical studies in nine families.	2001-05	11360275
Biochemical characterization and surfactant properties of horse allergens.	2001-05	11358533
Comparison of the sialic acid binding activity of transmissible gastroenteritis coronavirus and E. coli K99.	2001-05	11311429
Chemical derivatization of amino acids for in situ analysis of Martian samples by gas chromatography.	2001-04-27	11358249
Biochemical engineering of the side chain of sialic acids increases the biological stability of the highly sialylated cell adhesion molecule CEACAM1.	2001-04-27	11322763
A new fluorescent assay for sialyltransferase.	2001-04-23	11398988
Thermal stability of the hemagglutinin-neuraminidase from Sendai virus evidences two folding domains.	2001-04-20	11322945
Characterization of high-affinity binding between gangliosides and amyloid beta-protein.	2001-04-15	11368158
Corynebacterium diphtheriae surface proteins as adhesins to human erythrocytes.	2001-04-13	11313140
[The latest tumor marker in gastric cancer].	2001-04	11424436
Sialyltransferase activity in normal and atherosclerotic human aorta intima.	2001-04	11403646
Evaluation of recurrence in 36 subacute thyroiditis patients managed with prednisolone.	2001-04	11334386
Novel erythropoiesis stimulating protein (NESP) for the treatment of anaemia of chronic disease associated with cancer.	2001-04	11308271
A dose-finding and safety study of novel erythropoiesis stimulating protein (NESP) for the treatment of anaemia in patients receiving multicycle chemotherapy.	2001-04	11308270
Pharmacokinetics of novel erythropoiesis stimulating protein (NESP) in cancer patients: preliminary report.	2001-04	11308269
Development and characterization of novel erythropoiesis stimulating protein (NESP).	2001-04	11308268
Investigating the paradox of hypothyroidism and increased serum thyrotropin (TSH) levels in Sheehan's syndrome: characterization of TSH carbohydrate content and bioactivity.	2001-04	11297605
Human FSH isoforms: carbohydrate complexity as determinant of in-vitro bioactivity.	2001-03-28	11306170
Multiple ligand binding sites on domain seven of human complement factor H.	2001-03	11367528
Modulating cell surface immunoreactivity by metabolic induction of unnatural carbohydrate antigens.	2001-03	11306351
Biochemical characterization of the soluble form of tumor antigen MUC1 isolated from sera and ascites fluid of breast and pancreatic cancer patients.	2001-03	11300473
Characterization of blood group ABO(H)-active gangliosides in type AB erythrocytes and structural analysis of type A-active ganglioside variants in type A human erythrocytes.	2001-02-16	11342254
The interaction of selective plant lectins with neuraminidase-treated and untreated IgA1 from the sera of IgA nephropathy patients.	2001-02	11419909
Biosynthesis of N-acetylneuraminic acid in cells lacking UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase.	2001-02	11308027
Glycoproteins from insect cells: sialylated or not?	2001-02	11308014
Post genome analysis of Campylobacter jejuni.	2001	11422559
Castration-induced hyperactivity of seminal vesicle in the catfish Clarias batrachus: a case of paradox and blockade by antiandrogen (cyproterone acetate) treatment.	2001	11396845
Characterizing glycosylation pathways.	2001	11387039
Electrophoretic and aggregation behavior of bovine, horse and human red blood cells in plasma and in polymer solutions.	2001	11381164

Patents

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 Created by `admin` on `Mon Mar 31 18:04:51 GMT 2025` Edited by `admin` on `Mon Mar 31 18:04:51 GMT 2025`
Record UNII	GZP2782OP0
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

ACETYLNEURAMINIC ACID

Preferred Name

English

ACENEURAMIC ACID

Official Name

English

5-N-ACETYLNEURAMINIC ACID

Common Name

English

aceneuramic acid [INN]

Common Name

English

ACENEURAMIC ACID [USAN]

Common Name

English

SIALIC ACIDS N-ACETYLNEURAMINIC ACID

Common Name

English

(2S,4S,5R,6R)-5-ACETAMIDO-2,4-DIHYDROXY- 6-((1R,2R)-1,2,3-TRIHYDROXYPROPYL)OXANE-2-CARBOXYLIC ACID

Systematic Name

English

N-ACETYLNEURAMINIC ACID [USP-RS]

Common Name

English

UX-001

Code

English

N-ACETYLSIALIC ACID

Common Name

English

D-GLYCERO-D-GALACTO-NONULOSONIC ACID, 5-ACETAMIDO-3,5-DIDEOXY-

Common Name

English

Aceneuramic acid [WHO-DD]

Common Name

English

UX001

Code

English

(-)-5-ACETAMIDO-3,5-DIDEOXY-D-GLYCERO-D-GALACTO-NONULOSONIC ACID

Common Name

English

LACTAMINIC ACID

Common Name

English

N-ACETYLNEURAMINIC ACID [MI]

Common Name

English

D-GLYCERO-D-GALACTO-2-NONULOSONIC ACID, 5-(ACETYLAMINO)-3,5-DIDEOXY-

Common Name

English

N-ACETYLNEURAMIC ACID

Common Name

English

ACENEURAMIC ACID [JAN]

Common Name

English

N-ACETYL-D-NEURAMINIC ACID

Common Name

English

SIALIC ACID

Common Name

English

5-N-ACETYL-D-NEURAMINIC ACID

Common Name

English

SIALIC ACIDS N-ACETYLNEURAMINIC ACID [MI]

Common Name

English

NSC-111756

Code

English

N-ACETYLNEURAMINIC ACID

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

351511