Overview:
Gaucher disease, also known as lysosomal storage disorder is an inherited, rare disease that occurs due to less glucocerebrosidase (GCase) an enzyme which breaks down glucocerebroside building up around bone marrow, liver and spleen. Gaucher disease usually occurs in 1 in 40,000 to 60,000 newborn babies, where the lowest rate is 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, availability of companion diagnostic kits, usage of new genome sequencing, proteomics, and research and development infrastructure.
Regulatory agencies have developed a special drug designation for the Orphan disease treatment (Orphan drug Act 1983) which will be vital for the growth and treatment of Gaucher disease. Approval of the FDA to the companion diagnostic kits will enhance the diagnosis of rare diseases and will be helpful in the overall growth of 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 fetal stage. Recent technological advancement 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 its ambit. Challenges in this scenario would be the number of drugs under the pipelines are few as the available genetic data for the proper understanding of the disease is limited. Occurrence of Gaucher disease is in substantially small patient population. Patient compliance during the treatment is a big hurdle as the treatment demands a long term commitment. Restraints regarding Gaucher disease therapeutics market are inaccurate process validation, ambiguous reimbursement policies. Process validation regarding the signaling pathways which 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 are very few, which in turn acts as hurdle in the growth of the Gaucher disease therapeutics market. Ambiguous reimbursement policies, which employs genetic discrimination in the cost coverage for rare diseases is the biggest hurdle for the expenditure in the healthcare domain.The biomarkers used for the diagnosis for the genetic diseases are not having the optimum structural configuration, which results in inappropriate diagnosis of the disease.
The Pipeline product for Gaucher disease in phase 3 is ELELSYO (Protalix/Pfizer), phase 2 product is Abcertin (ISU, Abxis). The products in preclinical studies and drug development is NP003 (Neuraltus Pharmaceuticals, Inc.). The other treatment areas which are employed for the Gaucher disease treatment are stem cell therapy and protein chaperons for e.g. NCGC00188758 (Merck Millipore), AT3375 (Amicus) etc.
The global Gaucher disease therapeutics market is segmented on the following bases:
Key players identified for global Gaucher disease therapeutics market include:
The major pharmaceutical players involved in enzyme replacement therapy are, Sanofi Genzyme (Cerdelga, Cerezyme, Empagliflozine), Shire (VPRIV), JCR Pharmaceuticals Co.Ltd (JR-101), Greenovation Biopharma (Moss-GBA), Protein folding stimulant is Belrose Pharma (Ambroxol-Zywie); whereas Lixte Biotechnology Holdings (LB201/205) is active in enzyme inhibitor business segment.
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