Gaucher Disease Treatment Market, by Disease Type, Therapy
Type, Distribution Channel and Geography - Global Industry
Insights, Trends, Outlook, and Opportunity Analysis, 2018-2026
Gaucher disease is an autosomal recessive inherited metabolism disorder where a type of fat (lipid)
called glucocerebroside is unable to degrade. Body synthesis enzyme called glucocerebrosidase,
which breakdowns and reprocesses glucocerebroside. Gaucher disease is caused by mutations of a
single gene called GBA, which leads to very low levels of glucocerebrosidase enzyme leading to
low degradation of glucocerebroside. There are three types of Gaucher disease namely: type 1, type
2, and type 3. Type 1 is the most common type of Gaucher disease while Type 2 and 3 are not as
common as type 1.
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Approval of novel drugs is expected to boost growth of Gaucher disease treatment market
U.S. Food and Drug Administration (FDA) has approved multiple drugs in 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 FDA for type 1 Gaucher disease. These drugs offer variety of
options based on their requirements, 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 strategic collaboration of regulatory bodies for the
development of novel innovative drugs is expected to boost the growth of Gaucher disease
treatment market in near future. Lack of awareness regarding the symptoms, diagnosis, and
treatment of disease among rural population is a major factor restraining market growth.