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Gaucher Disease Treatment Market Collaborations between Product Manufacturers Like Genzyme , Pfizer, Shire Human Genetics Therapies and Actelion Pharmaceuticals

10-16-2019 07:28 AM CET | Business, Economy, Finances, Banking & Insurance

Press release from: Coherent Market Insights

Gaucher Disease Treatment

Gaucher Disease Treatment

Gaucher disease is an autosomal recessive inherited metabolism disorder that is caused by mutations of a single gene called GBA, which leads to very low levels of glucocerebrosidase enzyme which result into degradation of glucocerebroside. They can be classified into type 1, type 2, and type 3. Out of which, type 1 is the most common type of Gaucher disease.

Approval of a novel drug is projected to propel the market growth of Gaucher disease treatment market

U.S. Food and Drug Administration (FDA) has approved multiple drugs in the last few years for the treatment of disease. In 2010, FDA approved Vpriv (velaglucerase alfa), an enzyme replacement therapy (ERT), for type 1 Gaucher disease. In 2012, Elelyso (taliglucerase alfa) and in 2014, Cerdelga (eliglustat) was approved by the FDA for type 1 Gaucher disease. These drugs offer a variety of options based on their requirements, which in turn is expected to boost the growth of the market.

In July 2017, The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) collaborated to promote the use of innovative approaches in the development of medicines for Gaucher disease. Such a strategic collaboration of regulatory bodies for the development of novel innovative drugs is expected to boost the growth of the Gaucher disease treatment market in the near future. On the other hand, lack of awareness regarding the symptoms, diagnosis, and treatment of disease among the rural population is a major factor hampering the market growth.

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Market segmentation

The Gaucher disease treatment market is segmented based on disease type, treatment type, and distribution channel.

Based On Disease Type

Type 1 Gaucher disease

Type 2 Gaucher disease

Type 3 Gaucher disease

Based On Therapy Type

Enzyme Replacement Therapy (ERT)

Substrate Reduction Therapy (SRT)

Based On The Distribution Channel


Retail Pharmacies

Online Pharmacies

North America is projected to hold a dominant position owing to rising research activities and drug development for Gaucher disease

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North America is expected to hold the dominant position in the Gaucher disease treatment market owing to the rising number of research activities for the development of novel drugs. In 2016, the scientist from the National Institutes of Health researchers collaborated with the National Institute of Neurological Disorders and Stroke (NINDS) and the National Center for Advancing Translational Sciences (NCATS). Researchers identified a new molecule that offers a promising approach for the treatment of the rare Gaucher disease. Followed by Europe is expected to be the second-largest market for Gaucher disease treatment and this can be attributed to the initiative taken by European organizations to spread awareness regarding Gaucher disease among the population. For instance, in 2014, the European Gaucher Alliance launched Gaucher Awareness Day aims to spread awareness about this rare disease among the European population.

Key players operating in the global Gaucher disease treatment market include Genzyme Corporation, Pfizer, Inc., Shire Human Genetic Therapies, Inc., and Actelion Pharmaceuticals Ltd. (acquired by Johnson & Johnson in June 2017). There are various drugs in the pipeline of companies such as Lixte Biotechnology Holdings Inc, JCR Pharmaceuticals Co Ltd, Pharming Group NV, and Orphazyme ApS, for Gaucher disease treatment.

In-Depth TOC with Percentage Discount @

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Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

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