Pompe Disease - A Pipeline Analysis Report

Overview of the drug development pipeline for Pompe disease

Pompe disease is an autosomal recessive disorder and is also known as acid maltase deficiency disease or type II glycogen storage disease. Patients suffering from this disease experience glycogen accumulation primarily in skeletal and cardiac muscle, which is resulted from α-glucosidase (GAA) deficiency in lysosomes. The accumulation of glycogen impairs the ability of muscles to function properly. A nationwide newborn screening program for Pompe disease was started in Taiwan with high rate of successful results. Technavio’s market research analysts have predicted the introduction of such screening programs as one of the most promising approach to diagnose Pompe disease.

According to this pipeline analysis report, most of the drug development molecules in the pipeline are under the pre-clinical drug development stage. Our market research analysts have also identified that a small amount of drug molecules that are being developed comes under the phase III development stage. The pipeline landscape in the pipeline analysis report mentions the percentage of drug development molecules that are under the phase I/II and discovery stage.

Companies covered

This pipeline analysis report provides a detailed analysis of the companies that are actively involved in the development of drugs for the treatment of Pompe 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 –

• Pharming
• OXYRANE
• Etubics

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

• IV
• Oral
• IV + Oral

IV route of administration (ROA) involves the administration of drugs directly into the vein. According to this pipeline analysis report, the majority of drugs in the pipeline are being developed for IV administration.

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

• Recombinant enzyme
• Gene therapy
• Small molecule

Recombinant enzyme is produced by the modification of gene sequences and it generates useful therapeutic products. According to this pipeline analysis report, the majority of molecules that are currently in the drug development pipeline for Pompe disease are being developed as recombinant enzymes.

Key questions answered in the report include

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