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.
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.
Gaucher Disease Treatment Market Taxonomy
The Gaucher disease treatment market is segmented on the basis of disease type, treatment type, and distribution channel.
On the basis of disease type, the Gaucher disease treatment market is segmented into:
- Type 1 Gaucher disease
- Type 2 Gaucher disease
- Type 3 Gaucher disease
On the basis of therapy type, the Gaucher disease treatment market is segmented into:
- Enzyme Replacement Therapy (ERT)
- Substrate Reduction Therapy (SRT)
On the basis of distribution channel, the Gaucher disease treatment market is segmented into:
- Retail Pharmacies
- Online Pharmacies
North America is expected to hold dominant position due to increasing research activities and drug development for Gaucher disease
North America is expected to hold the dominant position in the Gaucher disease treatment market due to increasing research activities for the development of novel drugs. In 2016, the scientist from National Institutes of Health researchers collaborated with National Institute of Neurological Disorders and Stroke (NINDS) and the National Center for Advancing Translational Sciences (NCATS). Researchers identified a new molecule that shows promising approach for treatment of the rare Gaucher disease. Europe is expected to be the second largest market for Gaucher disease treatment due to the initiative taken by European organizations to spread awareness about Gaucher disease among the population. For instance in 2014, European Gaucher Alliance launched Gaucher Awareness Day aiming to spread awareness about this rare disease among the European population.
Key players operating in global Gaucher disease treatment market include Genzyme Corporation, Pfizer, Inc., Shire Human Genetics Therapies, Inc., and Actelion Pharmaceuticals Ltd. (acquired by Johnson & Johnson in June 2017). There are various drugs in pipeline of companies such as Lixte Biotechnology Holdings Inc, JCR Pharmaceuticals Co Ltd, Pharming Group NV and Orphazyme ApS, for Gaucher disease treatment.