aceneuramic acid

Action and Spectrum

Actions and Spectrum: 

aceneuramic acid works by providing a source of sialic acid, a crucial component in the production and maintenance of muscle and nerve cells. In individuals with HIBM, sialic acid is deficient due to a genetic mutation, which leads to muscle weakness and atrophy. aceneuramic acid aims to address this deficiency by supplementing sialic acid and potentially improving muscle function. 

The spectrum of aceramic acid’s action primarily focuses on treating the symptoms associated with HIBM, including muscle weakness and impaired mobility. It does not cure or modify the underlying genetic mutation causing the condition. 

Drug Interaction

Adverse Reaction

Frequency not defined 

Headache 

Muscle weakness 

Elevated liver enzymes 

Swelling 

Rash 

Dizziness 

Black Box Warning

Black Box Warning: 

None 

Contraindication / Caution

Pregnancy / Lactation

Pregnancy consideration:  

Pregnancy Category: N 

Breastfeeding warnings:  

Not available 

Pregnancy category: 

Category A: well-controlled and satisfactory studies show no risk to the fetus in the first or later trimester. 

Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women. 

Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.    

Category D: adequate data with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.    

Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.    

Category N: No data is available for the drug under this category. 

Pharmacology

Pharmacology: 

aceneuramic acid acts as an exogenous source of sialic acid, a critical component involved in producing and maintaining muscle and nerve cells. In individuals with HIBM, sialic acid is deficient due to a genetic mutation, leading to muscle weakness and atrophy. aceneuramic acid is believed to address this deficiency by supplying the required sialic acid to muscle and nerve cells. 

Pharmacodynamics:  

Pharmacokinetics: 

Absorption 

aceneuramic acid is administered orally as extended-release capsules. After ingestion, the capsules release the drug gradually, allowing for the absorption of sialic acid through the gastrointestinal tract. 

Metabolism and Elimination 

The metabolism and elimination of aceneuramic acid have yet to be fully characterized. However, sialic acid is a natural component of the body’s biochemical processes and is involved in various metabolic pathways. 

Adminstartion

Administration: 

aceneuramic acid is typically taken once daily. Follow the specific instructions provided by your healthcare provider regarding the timing of administration. Taking the medication with food improves absorption and reduces the risk of gastrointestinal side effects. 

Take the extended-release capsules whole with a glass of water. Do not chew, crush, or break the capsules, as it may interfere with the extended-release mechanism. 

Patient Information Leaflet

Patient information leaflet 

Generic Name: aceneuramic acid 

Why do we use aceneuramic acid?  

• Biomedical research: aceneuramic acid and its derivatives study sialic acid-related pathways, cellular interactions, and disease mechanisms. They help scientists better understand the role of sialic acids in health and disease. 
• Therapeutics: aceneuramic acid derivatives have shown potential therapeutic applications, particularly in disorders related to sialic acid metabolism. For example, a synthetic form of aceneuramic acid called N-acetyl-D-mannosamine (ManNAc) has been investigated to treat certain inherited metabolic disorders like hereditary inclusion body myopathy (HIBM) and GNE myopathy. 
• Glycoengineering: aceneuramic acid derivatives modify and engineer glycoproteins and glycolipids for various purposes. By manipulating sialic acid residues, researchers can alter these biomolecules’ properties, stability, and function for applications in biotechnology, diagnostics, and therapeutics.