Overview of the drug development pipeline for fabry disease
Fabry disease is a lipid-metabolism disorder or lysosomal storage disorder, inherited in an X-linked pattern. This disorder can lead to a deficiency of alpha-galactosidase A enzyme, which is responsible for breaking glycolipids a combination of sugar and lipid molecule. The deficiency of this enzyme can further lead to a build-up of GL-3/Gb3 and related glycolipids in the cells, which can later lead to life-threatening diseases such as kidney damage and dysfunction in the heart. Furthermore, the disease can also lead to heart attack and stroke. According to the NIH, in May 2018, around one out of 40,000-60,000 males are getting affected by Fabry disease every year, eventually, increasing the instances of the disease. Technavio’s market research analysts suggest, such rising instances of Fabry disease are expected to promote the drug development for Fabry disease in the forthcoming years.
According to this pipeline analysis report, most of the drug development molecules in the pipeline are being developed for the treatment of fabry 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 drug development for fabry 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 development molecules discontinued by companies.
Some of the companies covered in this pipeline analysis report are:
- Idorsia Pharmaceuticals
- JCR Pharmaceuticals
- Sanofi
Therapeutic assessment of the drug development pipeline for fabry disease by route of administration
- IV
- Oral
The intravitreal route of administration (ROA) involves the application of the drug directly into the vein, which will have a more direct effect on the target cells.
Therapeutic assessment of the drug development pipeline for fabry disease by therapy
- Monotherapy
- Combination therapy
According to this pipeline analysis report, all the molecules that are currently in the drug development pipeline for fabry 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 development molecules in the various development stages for fabry disease?
- What are the companies that are currently involved in the development of drug development molecules for fabry 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.