Market Insights
Fabry disease is a rare, X-linked, inherited disorder of lipid metabolism resulting from the deficient activity of the enzyme alpha-galactosidase-A. This enzyme is normally responsible for the breakdown of globotriaosylceramide, which results in abnormal deposits of a particular fatty substance in the walls of the blood vessels throughout the body. Characteristic features of Fabry disease include episodes of pain, cloudiness of the front part of the eye, clusters of small and dark red spots on the skin, and hearing loss.
The disease also involves life-threatening complications such as heart attack, kidney damage, and stroke. There are two types of Fabry disease: type 1 classic phenotype and type 2 later-onset phenotype. According to the National Organization for Rare Disorders (NORD), the incidence of type 1 classic phenotype is about 1 in 40,000 in males but varies with demography and race, ranging from about 1 in 18,000 to 1 in 95,000. The incidence of type 2 later-onset in males varies by ethnicity, demography, and race but is at least ten times more frequent than that of type 1 classic phenotype in the same region, ethnic group, or race.
There is no cure for Fabry’s disease, but medications are available to treat the condition. Enzyme Replacement Therapy (ERT) is used for treatment. Currently, only two ERT preparations, such as Replagal and Fabrazyme, are available on the market that is administered by intravenous infusion. In the USA, only Sanofi-Genzyme’s agalsidase beta (Fabrazyme) is FDA-approved. Shire’s Agalsidase alfa (Replagal) is approved in Europe and many other countries but not in the United States.
Fabrazyme and Replagal are administered every two weeks for a lifetime or until another appropriate medication become available. ERT is an extremely expensive treatment. Other treatments may also involve medications to reduce symptoms, such as pain medications, medications to treat stomach hyperactivity, blood thinners, and medications to manage arrhythmias and other heart disorders.
Key drivers for the global Fabry disease therapeutic market include extensive investigations and the adoption of advanced technology in research and development. Various therapeutic products, such as SMP-536, PRX-102, AT-1001, GC-1119, SAR-402671, PRX-102, and others, are in clinical trial studies. Fabry disease has been designated an ICD-10-CM code by the government and private insurance that can be used to indicate a diagnosis for reimbursement purposes, which would further drive the market. The disorder’s rare incidence rate (1:40,000) is a major impediment to growth in the global market for Fabry disease.
Companies such as Genzyme-Sanofi and Shire are the major players in the market, as only these companies have approved drugs on the market currently. Major players involved in the development of new medications include Amicus Therapeutics, Shire, Genzyme-Sanofi, Protalix, Sanofi-Aventis LLC, Novartis Pharmaceuticals, Pfizer, Bristol-Myers Squibb Company, GlaxoSmithKline plc, Amgen Inc., Teva Pharmaceutical Industries Ltd., Merck & Co., AbbVie Inc., Takeda Pharmaceutical Co. Ltd., and Green Cross Corp.
The Global Fabry Disease Therapeutic Market Is Divided Into the Following Subsegments:
- Global Fabry Disease Drug Market Revenue (USD Mn) By drug type, 2016–2024
- enzyme replacement therapy
- Alternative therapies
- Global Fabry Disease Drug Market Revenue (USD Mn), By Geography, 2016-2024
- North America
- US
- Canada
- Europe
- UK
- Germany
- Rest of Europe
- Asia-Pacific
- Japan
- China
- Rest of APAC
- Latin America
- Middle East and Africa
Key players identified for the global Fabry disease therapeutic market include:
Amicus Therapeutics, Shire, Genzyme-Sanofi, Protalix, Sanofi-Aventis LLC, Novartis Pharmaceuticals, Pfizer, Bristol-Myers Squibb Company, GlaxoSmithKline plc, Amgen Inc., Teva Pharmaceutical Industries Ltd., Merc & Co., AbbVie Inc., Takeda Pharmaceutical Co. Ltd., Green Cross Corp.
This report offers:
- An overview of the global markets for Fabry disease therapeutics
- Market trends assessment for the period 2014-2024, including historical data for 2014 and 2015 and projections through 2024, with respective CAGRs from 2016 to 2024.
- Qualitative assessment tools such as market drivers, challenges, and future prospects
- Market competition examination tools such as market share analysis and fractal map evaluation
- Focus on each level of market segmentation based on product approvals, launches, and current and anticipated market dynamics.
- A general overview of the industry structure
