Fabry disease is also known as Anderson-Fabry disease and alpha-galactosidase A deficiency. It is a rare genetic disorder of lipid metabolism resulting from the deficient activity of the alpha-galactosidase A (a-Gal A) enzyme. The deficiency of the enzyme is caused by the alterations in the genes that instructs the cells to make alpha-galactosidase A (a-Gal A) enzyme. Fabry disease is known to cause variety of systemic symptoms and complications, one of them being metabolic disorders. The metabolism of sphingolipids is minimized, in fabry disease which results in the multiple organ failure and death. Owing to the deficiency of alpha galactosidase A enzyme, glycolipid called Gb3 accumulates in the blood vessels and other organs and thus results in formation of blockage or clots that leads to malfunctioning of the system.
Fabry Disease Market: Drivers and Restraints
The major drivers that drive the fabry disease market growth are extensive R&D activities, adoption of advanced technologies in the research practices. Due to hereditary nature and severity of the disease, complications in physiological systems, such as cardiac system, urinary system and others are expected to increase the demand for trustworthy treatment. Extensive R&D practices is one of the valuable drivers of the market. Due to this, various therapeutic products, such as, PRX-102, JR-051, NP-003, GC-1119 and others, are under pipeline studies. However, rare incidence rate (1 in 40,000), lack of awareness about advancements in genetic sciences, technological limitations in certain region of the world are some of the restraining factors that may affect the growth of the fabry disease market.
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Fabry Disease Market: Segmentation
The global fabry disease market has been classified on the basis of treatment, end use and geography.
Based on treatment, the global fabry disease market is divided into following:
- Enzyme replacement therapy
- Gene therapy
- Pharmaceutical formulations containing agalsidase alpha
Based on end user type, the global fabry disease market is divided into following:
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Fabry Disease Market: Overview
The enzyme replacement therapy is designed to provide enzyme to the patients suffering from enzyme deficiency. It was found that this treatment was not a complete cure though it improved the metabolism. Enzyme replacement therapy is the most expensive and unaffordable treatment among the treatments for fabry disease, hence the market for enzyme replacement therapy is expected to register moderate growth in overall fabry disease market. The analgesics, anticonvulsants and NSAIDs are generally known for the treatment of pain and other symptoms associated with fabry disease hence, contributing majorly to the market.
Fabry Disease Market: Regional Overview
Geographically, the global fabry disease market is classified into regions namely, North America, Latin America, Western Europe, Eastern Europe, APAC, Japan, Middle East and Africa. North America is estimated to be the most lucrative region owing to the affordability of the treatment followed by Europe. The rare incidences in APAC and MEA countries are expected to limit the growth of fabry disease market in these regions.
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Fabry Disease Market: Key Players
The major players operating in this market and involved in development of new medications include Sanofi-Aventis LLC, iBio, Inc., GlaxoSmithKline plc, Genzyme Corporation, and Neuraltus Pharmaceuticals, Inc. Some of the existing key players in the global fabry disease market are Novartis Pharmaceuticals, Merc & Co., Inc., Bristol-Myers Squibb Company, AbbVie Inc., Amgen Inc., Teva pharmaceutical Industries Ltd., Pfizer Inc., Takeda Pharmaceutical Co. Ltd.