Gaucher disease, also known as lysosomal storage disorder, is an inherited, rare disease that occurs due to low levels of glucocerebrosidase (GCase), an enzyme that breaks down glucocerebroside that builds up around the bone marrow, liver, and spleen. Gaucher disease usually occurs in 1 in 40,000 to 1 in 60,000 newborn babies, with the lowest rate observed in the Asia Pacific and higher rates among Eastern European populations.
The major factorial entities supporting the growth in demand for Gaucher disease treatment are regulatory policies, the availability of companion diagnostic kits, the usage of new genome sequencing and proteomics technologies, and research and development infrastructure.
Regulatory agencies have developed a special drug designation for orphan disease treatment (Orphan Drug Act 1983), which will be vital for the growth and treatment of Gaucher disease. Approval by the FDA of the companion diagnostic kits will enhance the diagnosis of rare diseases and be helpful in the overall growth of the Gaucher disease therapeutic market.
Technological advancement in new genome sequencing will be helpful in the proper understanding of Gaucher disease at the genomic level and at the foetal stage. Recent technological advancements in the field of proteomics will be helpful in understanding the drug effect at the desired gene site, which will be helpful in developing drug delivery techniques like enzyme replacement therapy, substrate reduction therapy, etc. Investment by big pharmaceutical players in the research and development infrastructure in developing countries will bring the diagnosis and treatment of Gaucher disease under their ambit.
Challenges in this scenario would be the limited number of drugs in the pipeline and the limited genetic data available for a proper understanding of the disease. Gaucher disease affects a small percentage of the patient population.Patient compliance during the treatment is a big hurdle, as the treatment demands a long-term commitment. Restraints regarding the market for Gaucher disease therapeutics are inaccurate process validation and ambiguous reimbursement policies.
Process validation regarding the signalling pathways that are linked to Gaucher disease needs to be understood exhaustively, and the interpretation of the results in the diagnosis and treatment of Gaucher disease is still a burden as the number of trained healthcare professionals is very small, which in turn acts as a hurdle in the growth of the Gaucher disease therapeutics market. Ambiguous reimbursement policies, which employ genetic discrimination in the cost coverage for rare diseases, are the biggest hurdle for expenditure in the healthcare domain. The biomarkers used for the diagnosis of genetic diseases do not have the optimal structural configuration, which results in an inappropriate diagnosis of the disease.
The pipeline product for Gaucher disease in phase 3 is ELELSYO (Protalix/Pfizer); the phase 2 product is Abcertin (ISU, Abxis). The product in preclinical studies and drug development is NP003 (Neuraltus Pharmaceuticals, Inc.). The other treatment areas that are employed for Gaucher disease treatment are stem cell therapy and protein chaperons, e.g., NCGC00188758 (Merck Millipore), AT3375 (Amicus), etc.
The global Gaucher disease therapeutics market is segmented on the following basis:
- Enzyme Replacement Therapy (ERT)
- Substrate Reduction Therapy (SRT)
- North America
- Rest of Europe
- Asia Pacific
- South Korea
- South-east Asia
- Rest of Asia Pacific
- Latin America
- Rest of Latin America
- Middle East & Africa
- GCC Countries
- South Africa
- Rest of the Middle East and Africa
Key players identified for the global Gaucher disease therapeutics market include:
The major pharmaceutical players involved in enzyme replacement therapy are Sanofi Genzyme (Cerdelga, Cerezyme, and Empagliflozine), Shire (VPRIV), JCR Pharmaceuticals Co., Ltd. (JR-101), Greenovation Biopharma (Moss-GBA), Belrose Pharma (Ambroxol-Zywie), and Lixte Biotechnology Holdings (LB201/205), which is active in the enzyme inhibitor business segment.
This report offers:
- an overview of the global markets for Gaucher disease therapeutics.
- Market trends assessment for the period 2014-2024, including historical data for 2014 and 2015 as well as projections through 2024, with respective CAGRs from 2016 to 2024.
- Qualitative assessment tools such as market drivers, challenges, and future prospects
- Market competition examination tools such as market share analysis and fractal map evaluation
- Focus on each level of market segmentation based on product approvals, launches, and current and anticipated market dynamics.
- a general overview of the industry structure.
- Company profiles highlight key information about the major players operating in the global Gaucher disease therapeutics market.