Spinocerebellar Ataxia - A Pipeline Analysis Report

Overview of the drug development for spinocerebellar ataxia

It has been observed that spinocerebellar ataxia (SCA) is a hereditary disease caused due to mutations in the ATXN1 gene. Mutation in ATXN1 gene can lead to degenerative changes in the cerebellum and spinal cord. A person affected by the disease usually inherits the altered gene from an affected parent. The gene is passed from one generation to the next. According to the NIH, one to two per 100,000 people worldwide are affected by spinocerebellar ataxia. Spinocerebellar ataxia type 3, also called the Machado-Joseph disease (MJD), is an inherited form of ataxia that is characterized by the progressive clumsiness in the arms and legs. MJD is usually classified in three types including Type I, Type II, and Type III. Type I usually starts at the age of 10-30 years with faster progression and more dystonia and rigidity than ataxia. Type II is the most common type of MJD. It starts between the age of about 20 and 50 years and has an intermediate rate of progression.  Type III starts at the age of 40-70 years and progresses relatively slowly. Consequently, the rising incidences of the condition are further likely to boost the drug development for spinocerebellar ataxia in the next few years.

According to this pipeline analysis report, most of the drug molecules in the pipeline are being developed for spinocerebellar ataxia type 3. 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 spinocerebellar ataxia type 3. 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:

• Bioblast Pharma
• Biohaven Pharmaceutical
• Cadent Therapeutics
• Kissei Pharmaceutical
• Spark Therapeutics

Therapeutic assessment of the drug development for spinocerebellar ataxia by route of administration

• IV
• Oral
• Sublingual

The IV route of administration (ROA) application of the drug will have a more direct effect on the target cells.

Therapeutic assessment of the drug development for spinocerebellar ataxia by therapeutic modalities

• Small molecule
• Gene therapy
• Stem cell

According to this pipeline analysis report, a majority of the molecules that are currently in the drug development for spinocerebellar ataxia are being developed through small molecule therapeutic modalities 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 spinocerebellar ataxia?
• What are the companies that are currently involved in the development of drug molecules for spinocerebellar ataxia?
• 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.