Fabry Disease Treatment Market

Fabry Disease Treatment Market

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raksha

Author:

Raksha Sharma

Vineet

Fact-checked by:

Vineet Pandey

Shreyas

Editor:

Shreyas Tandon

Fabry Disease Treatment Market Outlook 2031

The global Fabry disease treatment market size was valued at USD 2.16 billion in 2022 and is projected to reach USD 4.81 billion by 2031, expanding at a CAGR of 9.3 % during the forecast period, 2023–2031. The market growth is attributed to the increasing incidence of the disease coupled with the increasing adoption of novel therapies such, as chaperone treatment.

Fabry disease is an X-linked lysosomal storage illness that causes progressive organ failure due to a lack of the alpha-galactosidase enzyme. An abnormal buildup of a specific fatty substance named globotriaosylceramide in numerous human tissues, including eyes, skin, kidneys, gastrointestinal tract, brain, heart, and central nervous system, causes this condition.

Fabry Disease Treatment Market Key Outlook

The Fabry disease does not have any viable treatments before enzyme replacement therapy, and the typical lifetime of a patient suffering from Fabry was up to 50 years. The disease's late onset and mild symptoms lead to several patients being undiagnosed.

Sanofi estimates that approximately 3,000 patients in the US suffer from Fabry disease. Patients with Fabry disease live longer lives on average than patients with other lysosomal storage disorders.


The market report finds that the COVID-19 pandemic has impacted the market significantly, as owing to the lockdown the supply chain system has been suspended. This has resulted in a limited healthcare workforce and limitations in outpatient services. Moreover, it has also impacted clinical research, for instance, according to the U.S. National Organization for Rare Disorders (NORD) 2020, nearly 98% of respondents were seriously concerned by the pandemic owing to various reasons. Among them, 95% of families were influenced by the pandemic, and more than 50% were having medical appointments replaced with telephone or video calls. The report further reveals that three out of five respondents expressed concerns about a shortage of medical supplies.

Fabry Disease Treatment Market Dynamics

Major Drivers 

Increasing R&D activities and the potential approval of promising pipeline drugs such as substrate reduction and enzyme replacement therapies are expected to propel the market during the period.

For instance, on May 22, 2023, Sangamo Therapeutics, Inc. a genomic medicine company, announced that the U.S. Food and Drug Administration (FDA) 
has granted Fast Track Designation to isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease.


Moreover, rising strategic collaboration and licensing agreement between the companies is expected to fuel the market during the forecast period. For instance, on April 4, 2023, Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for treating rare diseases, collaborated with the National Alliance on Mental Illness (NAMI), the nation's largest grassroots mental health organization, to deliver new mental health resources to patients suffering from Fabry disease.

Existing Restraints

Lack of awareness about Fabry disease among individuals is expected to delay the treatment and diagnosis is expected to restrain the market. Patients with Fabry disease face various challenges which in turn affect the quality of life. Individuals not having Fabry disease are not aware of the seriousness of its symptoms and the difficulties faced by the same. Thereby restraining the market. 

Emerging Opportunities

Advancements in therapies as well as in clinical trials related to Fabry disease and early recognition are expected to create lucrative opportunities for the market. Therapies including plant-derived ERTs, gene therapy, and substate reduction are some of the examples. Moreover, early recognition is important to prevent complications related to the disease. For instance, cardiac involvement in Fabry disease and its early recognition helps to prevent the risks.

  • On June 21, 2023, AceLink Therapeutics’ Phase 2 trial testing AL1211 as a treatment for Fabry disease has been cleared to launch by the U.S. Food and Drug Administration (FDA). The trial will evaluate AL1211’s safety and pharmacological properties in men diagnosed with classic Fabry disease who are willing to switch from their standard enzyme replacement therapy.

Scope of Fabry Disease Treatment Market Report

The market report includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have also been included in the report.

Attributes

Details

Report Title

Fabry Disease Treatment Market - Global Industry Analysis, Growth, Share, Size, Trends, and Forecast

Base Year

2022

Historic Data

2016–2021

Forecast Period

2023–2031

Segmentation

Treatment (Enzyme Replacement Therapy, Chaperone Treatment, Substrate Reduction Therapy, and Others)

Regional Scope

Asia Pacific, North America, Latin America, Europe, and Middle East & Africa

Report Coverage

Company Share, Market Analysis and Size, Competitive Landscape, Growth Factors, and Trends, and Revenue Forecast

Key Players Covered in the Report

Sanofi; Takeda Pharmaceutical Company Limited.; Amicus Therapeutics, Inc.; ISU ABXIS.; JCR Pharmaceuticals Co., Ltd.; Protalix Biotherapeutics; Sumitomo Pharma Co., Ltd.; Idorsia Pharmaceuticals Ltd; AVROBIO, Inc.; eleva GmbH; Moderna, Inc.; and GC Biopharma corp.

Fabry Disease Market Segment Insights

Treatment Segment Analysis 

Based on treatment, the market is segmented into enzyme replacement therapy, chaperone treatment, substrate reduction therapy, and others. The enzyme replacement therapy (ERT) segment is expected to account for a key market share during the forecast period owing to strong sales of Fabrazyme and Replagal, as well as the potential approval of promising pipeline candidates. Moreover, Sanofi's Fabrazyme and Shire's Replagal have received European approval, only Fabrazyme has received approval in the US.

Clinical trials are focusing on improving the safety and efficacy profile of ERTs, as well as the development of innovative oral medicines that can replace intravenous infusions. ERT is the gold standard for illness management. Galafold, a drug developed by Amicus Therapeutics, was recently licensed in the US, Canada, the European Union, Japan, Australia, Israel, and South Korea, as the first oral chaperone medication for adult patients.

Fabry Disease Treatment Market Treatments

Regional Analysis

In terms of region, the Fabry disease treatment market is classified as Asia Pacific, North America, Latin America, Europe, and Middle East & Africa. Asia Pacific is anticipated to expand at an impressive CAGR during the forecast period. The regional market growth is attributed to increasing healthcare expenditure and improving infrastructure. Rising population in emerging countries such as India and China is expected to propel the market in the region.

North America is expected to hold a significant market share during the forecast period. The usage of the medicine Fabrazyme is very high in the region which is fueling the regional market. During 2022, the net income generated by Sanofi is nearly USD 8.5 Billion owing to the production and sales of Fabrazyme. Other factors include the approval of certain therapeutics and their usage in the region.

For instance, the approval of Galafold by the U.S. Food and Drug Organization (FDA) which is the first oral medicine for Fabry disease in adults, and its availability in the region is further boosting the market in the region.  

Fabry Disease Treatment Market Regions

Segments

The global Fabry disease treatment market has been segmented on the basis of

Treatment

  • Enzyme Replacement Therapy
  • Chaperone Treatment
  • Substrate Reduction Therapy
  • Others

Region

  • Asia Pacific
  • North America
  • Latin America
  • Europe
  • Middle East & Africa

Key Players

Competitive Landscape

Key players competing in the Fabry disease treatment market include Sanofi; Takeda Pharmaceutical Company Limited.; Amicus Therapeutics, Inc.; ISU ABXIS.; JCR Pharmaceuticals Co., Ltd.; Protalix Biotherapeutics; Sumitomo Pharma Co., Ltd.; Idorsia Pharmaceuticals Ltd; AVROBIO, Inc.; eleva GmbH; Moderna, Inc.; and GC Biopharma corp.

Some of these players are using several market strategies such as acquisitions, mergers, collaborations, partnerships, capacity expansion, and product launches to enhance their market shares, generate revenue, and raise their business production line in the coming years.

  • In May 2023, Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for rare diseases, and Protalix BioTherapeutics, Inc., a biopharmaceutical company focused on the development of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx, announced that the European Commission (EC) has granted marketing authorization to PRX102 (pegunigalsidase alfa) in the European Union (EU) for treating adult patients with Fabry disease.
     
  • In March 2022, CR Pharmaceuticals Co., Ltd. and Sumitomo Dainippon Pharma Co., Ltd. announced the conclusion of an agreement regarding a marketing alliance for Agalsidase Beta BS I.V. Infusion 5 mg [JCR] and Agalsidase Beta BS I.V. Infusion 35 mg [JCR], which are currently being sold by JCR for the treatment of Fabry disease. The agreement focuses on distributing the product to the market by fiscal year 2022.
  • In August 2021, Amicus Therapeutics, a patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases, announced the European Commission has approved Galafold (migalastat) for use in adolescents aged 12 to <16 years weighing ≥ 45 kg with a confirmed diagnosis of Fabry disease and who have an amenable mutation.
     

Fabry Disease Treatment Market Key Players

1. Executive Summary
2. Assumptions and Acronyms Used
3. Research Methodology
4. Fabry Disease Treatment Market Overview
  4.1. Introduction
     4.1.1. Market Taxonomy
     4.1.2. Market Definition
  4.2. Macro-Economic Factors
     4.2.1. Industry Outlook
  4.3. Fabry Disease Treatment Market Dynamics
     4.3.1. Market Drivers
     4.3.2. Market Restraints
     4.3.3. Opportunity
     4.3.4. Market Trends
  4.4. Fabry Disease Treatment Market - Supply Chain
  4.5. Global Fabry Disease Treatment Market Forecast
     4.5.1. Fabry Disease Treatment Market Size (US$ Mn) and Y-o-Y Growth
     4.5.2. Fabry Disease Treatment Market Size (000’ Units) and Y-o-Y Growth
     4.5.3. Fabry Disease Treatment Market Absolute $ Opportunity
5. Global Fabry Disease Treatment Market Analysis and Forecast by Region
  5.1. Market Trends
  5.2. Introduction
     5.2.1. Basis Point Share (BPS) Analysis by Region
     5.2.2. Y-o-Y Growth Projections by Region
  5.3. Fabry Disease Treatment Market Size and Volume Forecast by Region
     5.3.1. North America
     5.3.2. Latin America
     5.3.3. Europe
     5.3.4. Asia Pacific
     5.3.5. Middle East and Africa (MEA)
  5.4. Absolute $ Opportunity Assessment by Region
  5.5. Market Attractiveness/Growth Potential Analysis by Region
  5.6. Global Fabry Disease Treatment Demand Share Forecast, 2019-2026
6. North America Fabry Disease Treatment Market Analysis and Forecast
  6.1. Introduction
     6.1.1. Basis Point Share (BPS) Analysis by Country
     6.1.2. Y-o-Y Growth Projections by Country
  6.2. North America Fabry Disease Treatment Market Size and Volume Forecast by Country
     6.2.1. U.S.
     6.2.2. Canada
  6.3. Absolute $ Opportunity Assessment by Country
  6.4. Market Attractiveness/Growth Potential Analysis
     6.4.1. By Country
     6.4.2. By Product Type
     6.4.3. By Application
  6.5. North America Fabry Disease Treatment Demand Share Forecast, 2019-2026
7. Latin America Fabry Disease Treatment Market Analysis and Forecast
  7.1. Introduction
     7.1.1. Basis Point Share (BPS) Analysis by Country
     7.1.2. Y-o-Y Growth Projections by Country
     7.1.3. Latin America Average Pricing Analysis
  7.2. Latin America Fabry Disease Treatment Market Size and Volume Forecast by Country
      7.2.1. Brazil
      7.2.2. Mexico
      7.2.3. Rest of Latin America
   7.3. Absolute $ Opportunity Assessment by Country
  7.4. Market Attractiveness/Growth Potential Analysis
     7.4.1. By Country
     7.4.2. By Product Type
     7.4.3. By Application
  7.5. Latin America Fabry Disease Treatment Demand Share Forecast, 2019-2026
8. Europe Fabry Disease Treatment Market Analysis and Forecast
  8.1. Introduction
     8.1.1. Basis Point Share (BPS) Analysis by Country
     8.1.2. Y-o-Y Growth Projections by Country
     8.1.3. Europe Average Pricing Analysis
  8.2. Europe Fabry Disease Treatment Market Size and Volume Forecast by Country
     8.2.1. Germany
     8.2.2. France
     8.2.3. Italy
     8.2.4. U.K.
     8.2.5. Spain
     8.2.6. Russia
     8.2.7. Rest of Europe
  8.3. Absolute $ Opportunity Assessment by Country
  8.4. Market Attractiveness/Growth Potential Analysis
     8.4.1. By Country
     8.4.2. By Product Type
     8.4.3. By Application
  8.5. Europe Fabry Disease Treatment Demand Share Forecast, 2019-2026
9. Asia Pacific Fabry Disease Treatment Market Analysis and Forecast
  9.1. Introduction
     9.1.1. Basis Point Share (BPS) Analysis by Country
     9.1.2. Y-o-Y Growth Projections by Country
     9.1.3. Asia Pacific Average Pricing Analysis
  9.2. Asia Pacific Fabry Disease Treatment Market Size and Volume Forecast by Country
     9.2.1. China
     9.2.2. Japan
     9.2.3. South Korea
     9.2.4. India
     9.2.5. Australia
     9.2.6. Rest of Asia Pacific (APAC)
  9.3. Absolute $ Opportunity Assessment by Country
  9.4. Market Attractiveness/Growth Potential Analysis
     9.4.1. By Country
     9.4.2. By Product Type
     9.4.3. By Application
  9.5. Asia Pacific Fabry Disease Treatment Demand Share Forecast, 2019-2026
10. Middle East & Africa Fabry Disease Treatment Market Analysis and Forecast
  10.1. Introduction
     10.1.1. Basis Point Share (BPS) Analysis by Country
     10.1.2. Y-o-Y Growth Projections by Country
     10.1.3. Middle East & Africa Average Pricing Analysis
  10.2. Middle East & Africa Fabry Disease Treatment Market Size and Volume Forecast by Country
     10.2.1. Saudi Arabia
     10.2.2. South Africa
     10.2.3. UAE
     10.2.4. Rest of Middle East & Africa (MEA)
  10.3. Absolute $ Opportunity Assessment by Country
  10.4. Market Attractiveness/Growth Potential Analysis
     10.4.1. By Country
     10.4.2. By Product Type
     10.4.3. By Application
  10.5. Middle East & Africa Fabry Disease Treatment Demand Share Forecast, 2019-2026
11. Competition Landscape
  11.1. Global Fabry Disease Treatment Market: Market Share Analysis
  11.2. Fabry Disease Treatment Distributors and Customers
  11.3. Fabry Disease Treatment Market: Competitive Dashboard
  11.4. Company Profiles (Details: Overview, Financials, Developments, Strategy)
     11.4.1. Sanofi Takeda Pharmaceutical Company Limited. Amicus Therapeutics, Inc. ISU ABXIS. JCR Pharmaceuticals Co., Ltd. Protalix Biotherapeutics Sumitomo Pharma Co., Ltd. Idorsia Pharmaceuticals Ltd AVROBIO, Inc. eleva GmbH Moderna, Inc. GC Biopharma corp.  

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FAQ Section

Some frequently asked quetions about this report!

Enzyme replacement therapy is the current treatment for Fabry disease.

Global manufacturer and pharmaceutical company Sanofi manufacture Fabrazyme

Drugs such as Fabrazyme and Galafold are approved by FDA

The cost of the enzyme replacement theory (ERT) is ranged around USD 40,941.1 to USD 69,176.5 per patient.