Lysosomal Storage Diseases Market to Witness Growth with the Emergence of Immunotherapy | Technavio

Technavio has published a new report on the global lysosomal storage diseases market from 2017-2021. (Graphic: Business Wire)

LONDON--()--Technavio’s latest market research report on the global lysosomal storage diseases (LSDs) market provides an analysis of the most important trends expected to impact the market outlook from 2017-2021. Technavio defines an emerging trend as a factor that has the potential to significantly impact the market and contribute to its growth or decline.

The global LSDs market is expected to grow at a CAGR of more than 7% during the forecast period because of the market penetration of the available branded drugs. The market is currently witnessing an increasing trend of using new applications for the development of safer and efficacious drugs to treat these diseases.

“The need for efficacious therapeutics that can either slow or reverse the progression is creating a huge demand in the market. The therapeutic options available to treat LSDs are very limited, especially in particular disease subsets such as Gaucher disease type II. Therefore, the lack of safe, effective, and cheap therapeutic options is creating a huge demand in the market. Any drug that could cater to these demands is expected to drive market growth,” says Sapna Jha, a lead analyst at Technavio for cardiovascular and metabolic disorders research.

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The top three emerging market trends driving the global lysosomal storage diseases market according to Technavio research analysts are:

  • Regulatory assistance in emerging nations
  • Increased R&D activities
  • Emergence of immunotherapy

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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. Countries like Taiwan and Korea have already implemented regulations regarding orphan drug designation while countries such as New Zealand and India are working on this.

“Emerging economies are increasingly becoming aware of the importance of orphan drug designations and are thereby working on the development of regulatory protocols required to award these designations. Also, these countries are also working on providing incentives such as reimbursements to pharmaceutical manufacturers to encourage research in this area,” says Sapna.

Increased R&D activities

R&D activities associated with LSDs are increasing because of the increase in mergers and acquisitions. Established pharmaceutical companies acquire later stage products from small companies, where efficacy has already been demonstrated, thereby cutting down initial R&D expenditure. For instance, in January 2014, Sanofi and Alnylam signed an agreement for the development of treatment therapies for rare genetic diseases. In February 2012, GlaxoSmithKline collaborated with Angiochem to promote the development and commercialization of EPiC-enzymes for the treatment of LSDs.

The collaborative treatment is aimed at developing LSDs treatments, which can address the neurological symptoms of this disease. In 2011, Sanofi acquired Genzyme, a company focused on the development of drugs for rare diseases including LSDs. In-licensing by established pharmaceutical companies, in turn, diverts funds to be spent by the smaller companies on marketing and commercialization to R&D development.

Emergence of immunotherapy

Immunotherapy enhances the innate powers of the immune system. The properties of the immune system to fight abnormal cells give immunotherapies a chance to successfully fight respiratory diseases, infectious diseases, cancer, rare diseases, and autoimmune disorders.

Recent advances preventing IgG production have been proven effective in both pre-clinical and clinical studies. As a means for general immune suppression, the drugs mycophenolate mofetil, cyclophosphamide, and methotrexate have been successfully used for the prevention of responses to ERT through inhibition of folic acid metabolism or as DNA alkylating agents. These agents prevent the formation of anti-GAA antibody in both pre-clinical and clinical studies, thereby demonstrating the therapeutic benefit of these agents in Pompe disease.

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Release Summary

The global lysosomal storage diseases market is expected to grow at a CAGR of more than 7% during the forecast period, 2017-2021.


Technavio Research
Jesse Maida
Media & Marketing Executive
US: +1 844 364 1100
UK: +44 203 893 3200