The following companies as key players in the global Lysosomal Storage Diseases Market: Actelion Pharmaceuticals, BioMarin, Genzyme, and Shire.

 

Other Prominent Vendors in the market are: Alexion Pharmaceuticals, Amicus Therapeutics, Chiesi Farmaceutici, Greenovation Biotech and FGK Clinical Research, Horizon Pharma, Leadiant Biosciences, Mylan, Pfizer, Protalix, Raptor, Recordati, Synageva BioPharma, Valerion Therapeutics, and Vtesse.

 

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Commenting on the report, an analyst from Technavio’s team said: “One trend in the market is regulatory assistance in emerging nations. The global LSDs market is expected to post a rapid CAGR, especially in emerging markets because of increasing legislative changes in these countries. These changes are aimed at promoting research and development for rare diseases.”

 

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According to the report, one driver in the market is unmet medical needs. The need for efficacious therapeutics that can either slow or reverse the progression of disease is on the rise in the market. The therapeutic options available to treat LSDs is very limited, especially in particular disease subsets such as Gaucher disease type II. The inability of the available treatments to improve neurological symptoms associated with LSDs because of the limited penetration of blood brain barrier (BBB) by ERTs also represent a high unmet need for this disease. For instance, there is a lack of safe treatment options for early stages of Pompe disease.

 

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Further, the report states that one challenge in the market is expensive treatment regimen for LSDs. The increasing cost of drugs has been a concern in the global LSDs market. The need for a cost-effective therapy to treat these disease, especially in developing countries is on the rise. The treatment of LSDs requires life-long administration of drugs, as it may lead to non-compliance in individuals and withdrawal from treatment regimens resulting in decreased consumption of medicines. The rise in cost of these drugs is due to lack of competition and low density of drugs marketed for the treatment of same indication.

 

The study was conducted using an objective combination of primary and secondary information including inputs from key participants in the industry. The report contains a comprehensive market and vendor landscape in addition to a SWOT analysis of the key vendors.

 

About Lysosomal Storage Diseases

 

Lysosomal storage diseases (LSDs) refer to a group of diseases arising because of the deficiency of lysosomal enzymes, which leads to the accumulation of partially digested or undigested macromolecules inside the cell. LSDs includes progressive diseases with variable ages of onset and clinical symptoms. LSDs are autosomal recessive disorders that are passed on from generation to generation. However, Fabry disease and MPS II are X-linked recessive disorders, which are inherited through mutated alleles on X chromosome.

 

Technavio’s analysts forecast the global lysosomal storage diseases market to grow at a CAGR of 7.43% during the period 2017-2021.

 

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