Gaucher Disease - A Pipeline Analysis Report

Overview of the drug development pipeline for Gaucher disease

Gaucher disease is caused by the mutations in the glucocerebrosidase protein. Some of the symptoms experienced by the patients suffering from this condition includes clotting of blood cells, enlarged liver and/or spleen, skeletal abnormalities, and low levels of circulating red blood cells. This disease is classified as Type 1, Type 2, and Type 3 Gaucher disease. It has been observed that the prevalence of Gaucher disease was maximum in Ashkenazi Jewish population in 2014. Technavio’s market research analysts have predicted that with the introduction of enzyme replacement therapy and substrate reduction therapy as some of the most promising approaches to treat Gaucher disease, the global Gaucher disease market will witness growth in the forthcoming years.

According to this pipeline analysis report, most of the drug development molecules in the pipeline are under the pre-clinical development stage. For instance, sponsors such as ADIENNE Pharma developed AND-LYS and AMO PHARMA developed RND-001, which are under pre-clinical stage. Our market research analysts have also identified that a small amount of drug molecules that are being developed comes under the phase I, phase II, phase I/II, IND, and some undisclosed development stages.

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 Gaucher 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 –

• ADIENNE
• AVROBIO
• Bionaturis

Therapeutic assessment of the drug development pipeline for Gaucher disease by preferred route of administration

• Intravenous
• Oral

The intravenous route of administration (ROA) involves the administration of drug substances directly into a vein. It has been observed that the majority of total therapeutics are being developed for intravenous administration.

Therapeutic assessment of the drug development pipeline for Gaucher disease by therapeutic modality

• Small molecule
• Recombinant enzyme

According to this pipeline analysis report, the majority of molecules that are currently in the drug development pipeline for Gaucher disease are being developed as recombinant enzyme. Recombinant enzyme is a manipulated form of enzyme, which is produced by modification of gene sequences and generate useful therapeutic products.

Key questions answered in the report include

• What are the drug development molecules in the various development stages for Gaucher disease?
• What are the companies that are currently involved in the development of drug development molecules for Gaucher 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.