Gaucher Disease Treatment Market Scope
Gaucher disease is a rare autosomal recessive metabolic disorder, with a prevalence rate of 1 in 100,000 individuals. It is the most common lysosomal storage disorder with Genetic conditions which are caused by mutations in the GBA (Glycosylceramidase Beta) Gene. This mutation leads to reduced production of glucocerebrosidase and a consequent accumulation of glucocerebroside. The Excess lipid accumulates in the brain, Bone marrow, Lungs, Kidney, Liver, and Spleen, which obstructs normal body functions. High dose is prescribed to them for the disease once a week, but, in some cases, a medium dose of medicine is given every week or three times in low dose quantity. However, the increasing prevalence of this disease in all age men & women and the subsequent increase in the number of drugs available in the market is anticipated to propel the market growth.
Attributes | Details |
---|
Study Period | 2018-2030 |
Base Year | 2023 |
Largest Market | North America |
Unit | Value (USD Million) |
Key Companies Profiled | Genzyme Corporation (United States), Shire (United States), Pfizer Inc. (United States), Johnson & Johnson Services, Inc.(United States), GlaxoSmithKline (GSK) PLC (United Kingdom), Eli Lilly and Company (United States), Alexion Pharmaceuticals Inc. (United States) and Anthera Pharmaceuticals, Inc. (United States) |
CAGR | 0.79% |
The market is a very fragmented market with very few players who rare strictly working for the development of the latest drug therapy. Most of the top players in the Gaucher disease drugs market are located in either Europe or North America. Research Analyst at AMA estimates that United States Players will contribute to the maximum growth of Global Gaucher Disease Treatment market throughout the predicted period.
Genzyme Corporation (United States), Shire (United States), Pfizer Inc. (United States), Johnson & Johnson Services, Inc.(United States), GlaxoSmithKline (GSK) PLC (United Kingdom), Eli Lilly and Company (United States), Alexion Pharmaceuticals Inc. (United States) and Anthera Pharmaceuticals, Inc. (United States) are some of the key players that are part of study coverage. Additionally, the Players which are also part of the research are Lixte Biotechnology Holdings Inc.(United States), JCR Pharmaceuticals Co Ltd.(Japan), Pharming Group NV (Netherlands) and Orphazyme ApS (Denmark).
About Approach
The research aims to propose a patent-based approach in searching for potential technology partners as a supporting tool for enabling open innovation. The study also proposes a systematic searching process of technology partners as a preliminary step to select the emerging and key players that are involved in implementing market estimations. While patent analysis is employed to overcome the aforementioned data- and process-related limitations, as expenses occurred in that technology allows us to estimate the market size by evolving segments as target market from the total available market.
Segmentation Overview
The study have segmented the market of Global Gaucher Disease Treatment market by Type and Region with country level break-up.
On the basis of geography, the market of Gaucher Disease Treatment has been segmented into South America (Brazil, Argentina, Rest of South America), Asia Pacific (China, Japan, India, South Korea, Taiwan, Australia, Rest of Asia-Pacific), Europe (Germany, France, Italy, United Kingdom, Netherlands, Rest of Europe), MEA (Middle East, Africa), North America (United States, Canada, Mexico). North America region held largest market share in the year 2023. Europe on the other hand stood as second largest market due to the presence of key companies into the region and high technological advancement.
Market Leaders and their expansionary development strategies
In 2021, Shire plc and Takeda Pharmaceutical Company Limited announced their merger, creating a company with a leading position in rare disease treatments, including Gaucher Disease.
In 2019, Pfizer received approval from the European Commission for ELELYSO (cerliponase alfa) for the treatment of Type 1 Gaucher Disease in adult and pediatric patients.
In July 2017, The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) collaborated to promote the use of innovative approaches in the development of medicines for Gaucher disease.
Influencing Trend:
Type I and type III being treatable by Enzyme Replacement Therapy (ERT) and ERT is seen Effective in Improving the Quality of Life of Patients
Market Growth Drivers:
Strategic Collaboration of Regulatory Bodies for the Development of Novel Innovative Drugs, Rising Demand for Substantial Treatment of the Disease and Increasing Number of People with Brain Disorder
Challenges:
Lack of Awareness Regarding the Symptoms, Diagnosis, and Treatment among Rural Population
Restraints:
Heavily Expensive and Lifelong undertaking Treatment and Limited Clinical Trial Data due to Fewer Patients Getting Affected
Opportunities:
Growing Medical Advancement in Research and Development for Rare Disorders
Key Target Audience
Gaucher Disease Treatment Manufacturers, Government and Regulatory Bodies, Healthcare Facilities, Pharmaceutical Companies, Potential Investors, Downstream Vendors and Traders/Suppliers/Distributors of Gaucher Disease Drugs