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


LONDON–(BUSINESS WIRE)–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

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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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