Fabry disease therapeutic market expected to Grow at 6.5% by 2031, 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 normally responsible for the breakdown of globotriaosylceramide, results in abnormal deposits of a particular fatty substance in walls of the blood vessel 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, hearing loss also involves life-threatening complications such as heart attack, kidney damage and stroke. Fabry disease can be divided into two types, type 1 classic phenotype and type 2 later-onset phenotype. According to National Organization for Rare Disorder (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 10 times more frequent than that of the type 1 classic phenotype from the same region, ethnic group or race.
There is no cure for Fabry disease, but medications are available treat the condition. Enzyme Replacement Therapy (ERT) is used for treatment. Currently only two ERT preparations such as Replagal and Fabrazyme are available in the market that are 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 is administered after every two weeks for lifetime or until another appropriate medication becomes available. ERT is extremely expensive treatment. Other treatment may also involve medications to reduce symptoms such as pain medications, medications to treat stomach hyperactivity, blood thinners and medication to manage arrhythmias and other heart disorders.
Key drivers for the global fabry disease therapeutic market includes, extensive investigations and adoption of advance technology in research and development. Various therapeutic products, such as, SMP-536, PRX-102, AT-1001, GC-1119, SAR-402671, PRX-102 and other are in clinical trial studies. Fabry disease has been designated ICD-10-CM code by government and private insurance that can be used to indicate diagnosis for reimbursement purpose, which would further drive the market. Major barriers of growth for the global for Fabry disease market is rare incidence rate of the disorder (1:40,000).
Companies such as Genzyme-Sanofi and Shire are the major player in market as only these company have approved drugs in the market currently. Major players involved in 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., Green Cross Corp. And Other Notable Players.
This report offers:
Company profiles highlighting key information about the major players operating in the Fabry Disease Therapeutics Market
Historical & Forecast Period
This study report represents analysis of each segment from 2022 to 2032 considering 2023 as the base year. Compounded Annual Growth Rate (CAGR) for each of the respective segments estimated for the forecast period of 2024 to 2032.
The current report comprises of quantitative market estimations for each micro market for every geographical region and qualitative market analysis such as micro and macro environment analysis, market trends, competitive intelligence, segment analysis, porters five force model, top winning strategies, top investment markets, emerging trends and technological analysis, case studies, strategic conclusions and recommendations and other key market insights.
Research Methodology
The complete research study was conducted in three phases, namely: secondary research, primary research, and expert panel review. key data point that enables the estimation of Fabry Disease Therapeutic market are as follows:
Market forecast was performed through proprietary software that analyzes various qualitative and quantitative factors. Growth rate and CAGR were estimated through intensive secondary and primary research. Data triangulation across various data points provides accuracy across various analyzed market segments in the report. Application of both top down and bottom-up approach for validation of market estimation assures logical, methodical and mathematical consistency of the quantitative data.
ATTRIBUTE | DETAILS |
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Research Period | 2022-2032 |
Base Year | 2023 |
Forecast Period | 2024-2032 |
Historical Year | 2022 |
Unit | USD Million |
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Drug Type
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Region Segment (2022-2032; US$ Million)
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Key questions answered in this report