According to Facts & Factors, the global Fabry disease treatment market size is expected to surpass around US$ 1.77 billion in 2019 to US$ 3.78 billion by 2026 and is expanding growth at a registered CAGR of 9.83% from 2020 to 2026.

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Market Overview:

Fabry disease is an extremely uncommon form of disease. A patient will experience progressive organ dysfunction if they do not have enough alpha-galactosidase enzyme in their body. The abnormal accumulation of a specific fatty matter called globotriaosylceramide is the primary factor that contributes to the development of Fabry diseases. This abnormal accumulation can have an effect on the skin, heart, eyes, brain, kidneys, central nervous system, and gastrointestinal system. It can also affect the central nervous system.

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Because males only have one copy of the X chromosome, they are more likely to be affected by the condition being discussed. On the other hand, the female population acts as carriers and is less likely to experience complications from the disease in its early stages. On the other hand, it is possible for neurological issues such as high blood pressure, heart failure, stroke, and other complications to develop. Burning sensations, excessive sweating, cloudy vision, irregular bowel movements, and other symptoms that are most common in this disorder are the primary symptoms. Other symptoms include those that are less common.

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The -gal enzyme found in the leukocytes of the patients is tested to determine its presence. When it comes to neonates, the presence of this disease can be determined through an examination to look for abnormalities in heart or kidney development. Because there is currently no treatment that can reverse the effects of Fabry disease, the focus of treatment is on reducing symptoms and minimising the risk of developing complications. Patients who present with only mild symptoms are frequently given the wrong diagnosis. In the coming years, there are a number of factors that are anticipated to contribute to the market expanding at a significant rate.

Key Market Participants:

ISU Abxis Co Ltd., Greenovation Biotech GmbH, Amicus Therapeutics Inc., Avrobio Inc., Shire Plc., Sanofi S.A., Moderna Therapeutics Inc., JCR Pharmaceuticals, Protalix Biotherapeutics Inc., and Idorsia Pharmaceuticals Ltd. are some of the major players that are active in the global Fabry disease treatment market.

Market Drivers:

  • It is anticipated that the presence of a robust drug pipeline for the treatment of Fabry disease will drive development. 

Novel drugs for the treatment of Fabry disease have been granted approval by a number of regulatory bodies in key regions in recent months. As an illustration, the Food and Drug Administration (FDA) of the United States of America granted approval in 2018 to the pharmaceutical product Galafold, which was developed by Amicus Therapeutics, Inc. (migalastat). It is the first oral drug that has been given the OK to treat Fabry disease, and it is specifically intended for adults.

In addition, market players are making massive investments in the production of novel therapies for the treatment of Fabry disease. Strategic expenditure by various market players will proliferate the growth of the global market in the years to come. For example, AvroBio Inc. announced in February 2018 that it had successfully completed a funding round of sixty million dollars to finance the phase 2 trial of AVR-RD-01, a novel gene therapy that is currently under development for the treatment of Fabry disease. Over the course of the period covered by the forecast, the growth of the market will be driven by these factors.

According to the National Fabry Disease Foundation, however, Fabry disease affects only one person out of every fifty thousand. In addition, the diagnosis of Fabry disease is confirmed by demonstrating a deficiency in an enzyme within the male population and locating the distinct GLA gene mutation within both females and males. As a consequence of this, low diagnostic rates in developing economies as a consequence of a lack of such diagnostic procedures and qualified professionals are anticipated to be a barrier to the expansion of the global market for Fabry disease treatments.

Impact of COVID-19 on the Worldwide Market for the Treatment of Fabry Disease

It is currently unknown what effect COVID-19 will have on Fabry disease patients who are undergoing enzyme replacement therapy. Because of concerns about spreading infection, the hospital staff had to temporarily stop giving infusions to a few of the patients receiving care there. In other lysosomal storage diseases, the effects of temporarily interrupting treatment have been identified in greater detail. Despite this, resuming therapy does not completely reverse the clinical deterioration that was caused by temporarily interrupting treatment. In the event that enzyme replacement therapy is required during a pandemic, it appears that home therapy is the most effective way to maintain access to the treatment. It is anticipated that the growth of the market will be fueled by these factors over the course of the forecast period.

Regional Dominance:

  • It is anticipated that the North American market will dominate the expansion of the global Fabry disease treatment market.

In 2019, the market for treatments for Fabry disease was led by the North American region, which accounted for the largest market share worldwide. The growth of the region’s market is being fueled in part by an increase in the acceptance of novel therapies, as well as by favourable reimbursement policies and enhanced healthcare services. As a result of health insurance programmes covering the costs of medical expenses, pharmaceutical companies are increasing the amount of money they invest in research and development in the field of rare diseases.

Pharmaceutical companies were presented with a significant growth opportunity in Asia and the Pacific as a result of the region’s variously improved healthcare infrastructure and the rising healthcare spending in emerging economies. During the period covered by the forecast, it is anticipated that this region will hold a larger share of the market.

Additionally, the availability of biosimilars is anticipated to make the costs associated with these therapies more reasonable in this industry. According to the Indian Ministry of Health and Family Welfare, the average annual cost of enzyme replacement therapy in India was USD 26,334 in 2016. This figure was reported in 2016. These are the factors that are thought to be responsible for propelling demand in the region in the years to come.

The global Fabry disease treatment market is segmented as follows:

By Drugs:

  • Agalsidase Beta
  • Migalastat
  • Pipeline Drugs

By Treatment:

  • Enzyme Replacement Therapy (ERT)
  • Chaperone Treatment
  • Substrate Reduction Therapy (SRT)
  • Others

By Route of Administration:

  • Oral
  • Intravenous

By Region:

  • North America
    • The U.S.
    • Canada
  • Europe
    • France
    • The UK
    • Spain
    • Germany
    • Italy
    • Rest of Europe
  • Asia Pacific
    • China
    • Japan
    • India
    • South Korea
    • Southeast Asia
    • Rest of Asia Pacific
  • Latin America
    • Brazil
    • Mexico
    • Rest of Latin America
  • Middle East & Africa
    • GCC Countries
    • South Africa
    • Rest of Middle East & Africa

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