Gaucher disease is a type of a lysosomal storage disorder that often causes liver and spleen enlargement, bone issues and blood problems. The disease involves fatty lipid deposition in certain organs and bones. The disease is treated by bone marrow transplantation and enzyme replacement therapy (ERT). Gaucher disease is categorized into type I, II and III. According to the data published by National Organization for Rare Disorders (NORD), Gaucher disease type I is the most common type, accounting for more than 90% of cases. There are approximately 6,000 individuals with Gaucher disease in the U.S. Sanofi Genzyme is in the process of developing GZ402671, as a glucosylceramide synthase inhibitor for the treatment of Gaucher’s type III disease. Some of the companies having a pipeline of Gaucher disease include Sanofi Genzyme, Orphazyme ApS, Belrose Pharma, Inc. and others.
The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with detailed analysis of pipeline and clinical trials. Pipeline analysis of drugs by phases, includes product description and development activities, including information about clinical results, designations, collaborations, licencing, grants, technology and others.