About Fabry Disease
Fabry Disease is an X-linked genetic disease caused by a deficiency of the enzyme αgalactosidase A (α-Gal A) in the body. The enzyme α-Gal A’s function is to break down fatty element called globotriaosylceramide (or GL3). When α-Gal A is absent, GL3 builds up in the blood vessel walls in the body. As the unusual storage of GL3 increases with time, the body’s blood vessels become narrowed, which leads to decreased blood flow and undernourishment of the tissues. This abnormal process occurs in blood vessels throughout the body, particularly those blood vessels in the skin, kidneys, heart, brain and nervous system. The early symptoms of Fabry disease such as decreased sweating, heat intolerance, proteinuria, a reddish-purple skin rash, severe pains in the hands and feet, hearing loss, chronic fatigue, and gastrointestinal issues, generally start in childhood. Fabry disease symptoms affect both women and men.
Attributes | Details |
---|
Study Period | 2018-2030 |
Base Year | 2023 |
Unit | Value (USD Million) |
CAGR | 8.1% |
New and existing market players are coming up with better treatment approaches. The companies are exploring the market in new regions by adopting expansions, investments, new launches and collaborations as their preferred strategies. The players are exploring new geographies through expansions and acquisition to avail competitive advantage through combined synergies. Analyst at AMA Research estimates that United States Players will contribute the maximum growth to Global Fabry Disease market throughout the forecasted period. Established and emerging Players should take a closer view at their existing organizations and reinvent traditional business and operating models to adapt to the future.
Bristol-Myers Squibb Company (United States), AbbVie Inc. (United States), Teva pharmaceutical Industries Ltd. (Israel), Pfizer Inc. (United States), Takeda Pharmaceutical Co. Ltd. (Japan), Sanofi-Aventis LLC (United States), GlaxoSmithKline plc (United Kingdom), Shire (United States), Protalix BioTherapeutics (Israel), Green Cross Corp. (South Korea) and Idorsia Pharmaceuticals Ltd (Switzerland) are some of the key players that are part of study coverage.
Segmentation Overview
AMA Research has segmented the market of Global Fabry Disease market by , Application (Child, Women and Men) and Region.
On the basis of geography, the market of Fabry Disease has been segmented into South America (Brazil, Argentina, Rest of South America), Asia Pacific (China, Japan, India, South Korea, Taiwan, Australia, Rest of Asia-Pacific), Europe (Germany, France, Italy, United Kingdom, Netherlands, Rest of Europe), MEA (Middle East, Africa), North America (United States, Canada, Mexico). If we see Market by Treatment, the sub-segment i.e. Enzyme Replacement Therapy will boost the Fabry Disease market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth.
Influencing Trend:
Increasing Regulatory Assistance in Emerging Countries
Market Growth Drivers:
Growing Acceptance of Advanced Technologies in Research Practices and Special Provisions for Orphan Drugs
Challenges:
Technological Limitations for the Treatment
Restraints:
Lack of Awareness about Advancements in Genetic Sciences and Complications in Physiological Systems
Opportunities:
Increasing Investment on Healthcare Sector in Developing Countries and Special Incentives in the Form of Funding to Vendors to Conduct Research in Fabry Disease
Market Leaders and their expansionary development strategies
In August 2023, Sanofi Genzyme launched Fabrazyme (agalsidase beta) Infused Reconstituted Powder for Injection, a new formulation of their existing enzyme replacement therapy for Fabry Disease. This new formulation requires a shorter infusion time compared to the previous liquid formulation.
In September 2023, Amicus Therapeutics received FDA approval for Galafold (migalastat hydrochloride) capsules, a new oral treatment option for Fabry Disease patients with amenable GLA mutations. This was the first oral therapy approved for Fabry Disease in the US.
On 10 August 2018 The U.S. Food and Drug Administration have approved Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease.
Key Target Audience
Suppliers of Pharmaceuticals, Wholesalers, Distributors and Retailers of Pharmaceuticals, Healthcare Industry, Research Firms and Governmental Bodies
About Approach
To evaluate and validate the market size various sources including primary and secondary analysis is utilized. AMA Research follows regulatory standards such as NAICS/SIC/ICB/TRCB, to have a better understanding of the market. The market study is conducted on basis of more than 200 companies dealing in the market regional as well as global areas with the purpose to understand the companies positioning regarding the market value, volume, and their market share for regional as well as global.
Further to bring relevance specific to any niche market we set and apply a number of criteria like Geographic Footprints, Regional Segments of Revenue, Operational Centres, etc. The next step is to finalize a team (In-House + Data Agencies) who then starts collecting C & D level executives and profiles, Industry experts, Opinion leaders, etc., and work towards appointment generation.
The primary research is performed by taking the interviews of executives of various companies dealing in the market as well as using the survey reports, research institute, and latest research reports. Meanwhile, the analyst team keeps preparing a set of questionnaires, and after getting the appointee list; the target audience is then tapped and segregated with various mediums and channels that are feasible for making connections that including email communication, telephonic, skype, LinkedIn Group & InMail, Community Forums, Community Forums, open Survey, SurveyMonkey, etc.