Wilsons Disease - A Pipeline Analysis Report

Overview of the drug development pipeline for Wilson’s disease

Wilson’s disease, also known as hepatolenticular degeneration, is a rare genetic metabolic disorder in which excessive copper ions start accumulating in various body parts such as the liver, brain, and eyes. The tissues and organs get damaged due to the toxic effect of high levels of copper ions. Chronic liver diseases, central nervous system abnormalities, and psychiatric disturbances are some of the signs and symptoms of Wilson’s disease. Mutation of the ATP7B gene, which is responsible for encoding an ATP7B protein, is the primary cause of the disorder. The elimination of excessive copper ions through the liver is the normal function of the ATP7B protein. Due to the mutation in the gene, this protein does not function properly, thus resulting in the abnormal accumulation of copper ions in the body. People affected with Wilson’s disease go through a variety of neurologic signs and symptoms including tremors, muscle stiffness, problems with speech, swallowing, and physical coordination. According to the National Human Genome Research Institute, Wilson’s disease occurs in approximately 1 in 30,000 people globally. Nearly 1 in 90 people may be the carriers of the disease gene. In 2011, the global prevalence of Wilson’s disease was found to be between 1/30,000 and 1/100,000 individuals. Consequently, the growing incidences of the disease are further expected to promote the drug development for Wilson’s disease in the upcoming years.

According to this pipeline analysis report, most of the drug molecules in the pipeline are being developed for Wilson’s disease. Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.

Companies covered

This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of drug molecules for the treatment of Wilson’s disease. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the drug molecules discontinued by companies.

Some of the companies covered in this pipeline analysis report are –

• GMP-Orphan
• Vivet Therapeutics
• Ultragenyx Pharmaceutical
• Wilson Therapeutics

Therapeutic assessment of the drug development pipeline for Wilson’s disease by route of administration

• Oral
• Intravenous

The oral route of administration (ROA) involves the application of the drug directly into the mouth cavity, which will have a more direct effect on the target cells.

Therapeutic assessment of the drug development pipeline for Wilson’s disease by therapy

• Monotherapy

According to this pipeline analysis report, all the molecules that are currently in the gene therapy pipeline for Wilson’s disease are being developed as monotherapy drugs and most of these molecules are in the pre-clinical stage of development.

Key questions answered in the report include

• What are the drug molecules in the various development stages for Wilson’s disease?
• What are the companies that are currently involved in the development of drug molecules for Wilson’s disease?
• Insight into discontinued/inactive molecules with appropriate reasoning?
• What are the major regulatory authorities approving drugs in various regions?
• Detailed profiling of each active molecule

Technavio also offers customization on reports based on specific client requirement.