Friedreichs Ataxia - A Pipeline Analysis Report

Overview of the drug development pipeline for Friedreich’s ataxia

It has been observed that Friedreich’s ataxia is defined as a rare genetic disease caused by a defect (mutation) in a gene labeled FXN, which carries the genetic code for a protein called frataxin. Friedreich’s ataxia usually starts in childhood, and it worsens over a period. This also leads to impairments in muscle coordination. People with Friedreich’s ataxia can also have a form of hypertrophic cardiomyopathy, which enlarges and weakens the heart muscle. Some of the key symptoms of the disease include difficulty in walking and poor balance, shortness of breath, and loss of normal reflexes, especially for knees and ankles. Consequently, the rising incidences of Friedreich’s ataxia are expected to boost the drug development for Friedreich’s ataxia over the predicted period.

According to this pipeline analysis report, most of the drug molecules in the pipeline are being developed for Friedreich’s ataxia. 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 gene therapy molecules for the treatment of Friedreich’s ataxia. 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 –

• Adverum
• Agilis Biotherapeutics
• Bioblast Pharma
• Cardero Therapeutics
• Chondrial Therapeutics

Therapeutic assessment of the drug development pipeline for Friedreich’s ataxia by route of administration

• Oral

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

Therapeutic assessment of the drug development pipeline for Friedreich’s ataxia by therapy

• Monotherapy
• Combination therapy

According to this pipeline analysis report, most of the molecules that are currently in the drug development pipeline for Friedreich’s ataxia 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 Friedreich’s ataxia?
• What are the companies that are currently involved in the development of drug molecules for Friedreich’s 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.