arimoclomol (FDA Approval Pending)

Action and Spectrum

Actions and Spectrum  

arimoclomol is an inducer of heat shock proteins (HSPs). Heat shock proteins are a group of cellular chaperones that help maintain proper protein folding and prevent the accumulation of misfolded or aggregated proteins.

Drug Interaction

Adverse Reaction

None

Black Box Warning

Black Box Warning: 

None 

Contraindication / Caution

Contraindication/Caution:   

None  

Pregnancy / Lactation

Pregnancy warnings:     

Pregnancy category: N/A 

Lactation: Excretion into human milk is unknown  

Pregnancy Categories:         

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: There is no data available for the drug under this category.

Pharmacology

Pharmacology  

arimoclomol increases the synthesis of heat shock proteins (HSPs), which protect cells from damage, but only in cells that are physiologically stressed, such as those affected by illness.  

In order to repair cells and lessen protein misfolding and aggregation, it activates proteins that serve as pharmacologic chaperones. This enables the cells to handle accumulated lipid. 

Pharmacodynamics 

Patients with Niemann-Pick disease (NPD) type C disease are unable to transfer cholesterol and lipids within cells, resulting in an abnormal accumulation of these substances within a variety of bodily tissues, including the brain tissue. 

Pharmacokinetics 

Limited information available on ADME

Adminstartion

Administration  

arimoclomol is administered orally in the form of tablets or capsules. 

Patient Information Leaflet

Patient information leaflet 

Generic Name: arimoclomol (FDA Approval Pending) 

Why do we use arimoclomol? 

arimoclomol has been investigated in clinical trials for the treatment of Niemann-Pick disease Type C (NPD), a rare and progressive genetic disorder characterized by impaired lipid metabolism.   

arimoclomol has undergone clinical trials as a potential treatment for Amyotrophic Lateral Sclerosis (ALS). It is a progressive neurodegenerative disease affecting motor neurons.