Gurgaon Samachar

Fabry Disease Treatment Market

(Albany, USA) DelveInsight’s “Fabry Disease Market Insights, Epidemiology, and Market Forecast-2034” report delivers an in-depth understanding of Fabry Disease, historical and forecasted epidemiology as well as the Fabry Disease market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

The Fabry Disease market report provides current treatment practices, emerging drugs, the market share of tmarkets. The Report also covers current Fabry Disease treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the Fabry Disease market.

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Some facts of the Fabry Disease Market Report are:

• According to DelveInsight, Fabry Disease market size is expected to grow at a decent CAGR by 2034.
• As per DelveInsight analysis, the Fabry disease market size in the 7MM was approximately USD ~1700 million in 2024.
• As per the analysis conducted by DelveInsight, in 2022 the total diagnosed prevalent cases of Fabry disease was 15,290 in the 7MM and these cases are further projected to increase during the study period (2020-2034).
• Leading Fabry Disease companies working in the market are Protalix Biotherapeutics, Sanofi Genzyme, Sangamo Therapeutics, Freeline Therapeutics, 4D Molecular Therapeutics, Idorsia Pharmaceuticals, GREENOVATION BIOTECH GMBH, Shire, Takeda, Amicus Therapeutics, Protalix, uniQure, Codexis, MP6 Therapeutics, CellGenTech, and others.
• Key Fabry Disease Therapies expected to launch in the market are PRX-102 (Pegunigalsidase Alfa), Venglustat, ST-920, FLT190, 4D-310, Lucerastat, Moss-aGal, and others.
• In February 2025, Amicus Therapeutics presented oral sessions and posters highlighting its migalastat development programs at the 21st Annual WORLD Symposium.
• In January 2025 presentation, Idorsia Pharmaceuticals shared that they expect the Phase III Open-label Extension (OLE) study results in Q2 2025 and plan to discuss the regulatory pathway with the US FDA thereafter.
• In January 2025, Sanofi announced that the regulatory submission for venglustat for Fabry disease is anticipated in 2026.
• In December 2024, CHIESI Farmaceutici and Protalix BioTherapeutics announced that the EMA had validated the variation submission for pegunigalsidase alfa to label a less frequent dosing regimen at a dose of 2 mg/kg body weight administered every 4 weeks in adult patients with Fabry disease.
• In September 2024, UniQure Biopharma mentioned that with the support of the designation for its Phase I/IIa clinical trial, the company is eager to rapidly generate clinical proof-of-concept data and provide initial results in 2025.
• On March 2024, Sanofi announced results of a Phase 4 study which evaluates the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment with Fabrazyme.
• On April 2024, 4D Molecular Therapeutics announced results of an Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement

Fabry Disease Overview

Fabry Disease is a rare, inherited lysosomal storage disorder caused by a deficiency of the enzyme alpha-galactosidase A. Fabry Disease leads to the accumulation of globotriaosylceramide (Gb3) in various tissues, including the kidneys, heart, and nervous system. Fabry Disease symptoms can include chronic pain, angiokeratomas, heat intolerance, corneal opacities, and gastrointestinal issues. Fabry Disease diagnosis typically involves enzyme assay testing and genetic analysis to confirm mutations in the GLA gene. Fabry Disease primarily affects males, but female carriers can also exhibit Fabry Disease-related complications due to X-linked inheritance.

Fabry Disease progression can result in life-threatening complications such as Fabry Disease-induced renal failure, Fabry Disease-related cardiomyopathy, and Fabry Disease-associated stroke. Fabry Disease treatment includes enzyme replacement therapy (ERT) and pharmacological chaperones that help reduce Gb3 accumulation. Fabry Disease management also involves symptomatic care and regular monitoring of Fabry Disease biomarkers. Fabry Disease awareness is increasing due to advances in Fabry Disease research and clinical trials focused on gene therapy and novel treatment approaches. Early Fabry Disease screening can significantly improve patient outcomes. Fabry Disease specialists, including nephrologists, cardiologists, and geneticists, play a crucial role in personalized care. Comprehensive Fabry Disease education is essential for early detection, timely intervention, and better quality of life.

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Fabry Disease Market

The Fabry Disease market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Fabry Disease market trends by analyzing the impact of current Fabry Disease therapies on the market and unmet needs, and drivers, barriers, and demand for better technology.

This segment gives a thorough detail of the Fabry Disease market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Fabry Disease market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

The Fabry Disease market is driven by several key factors. Increasing awareness about rare genetic disorders and advancements in diagnostic technologies have led to earlier and more accurate diagnosis of Fabry Disease, facilitating timely treatment. The development of innovative therapies, such as enzyme replacement therapy (ERT) and pharmacological chaperones, has expanded the treatment landscape and improved patient outcomes, further propelling market growth. Additionally, rising healthcare expenditure, favorable reimbursement policies in developed countries, and growing investment in research and development by pharmaceutical companies contribute significantly to the market’s expansion. Strategic collaborations and regulatory support for orphan drugs also play a pivotal role in encouraging innovation and accessibility.

However, the Fabry Disease market faces notable barriers. The high cost of treatment, particularly ERT, poses a substantial financial burden on patients and healthcare systems, limiting access in low- and middle-income regions. Limited awareness and diagnostic capabilities in some parts of the world contribute to underdiagnosis or misdiagnosis, delaying treatment initiation. Moreover, the small patient population characteristic of rare diseases often results in limited clinical data and challenges in conducting large-scale clinical trials. These factors, along with stringent regulatory requirements and long development timelines, can hinder the timely introduction of new therapies into the market.

According to DelveInsight, the Fabry Disease market in 7MM is expected to witness a major change in the study period 2020-2034.

Fabry Disease Epidemiology

The Fabry Disease epidemiology section provides insights into the historical and current Fabry Disease patient pool and forecasted trends for seven individual major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Fabry Disease market report also provides the diagnosed patient pool, trends, and assumptions.

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As per DelveInsight’s assessments, in the 7MM Fabry Disease is more prevalent in males than in females but there are some differences at the regional level

Fabry Disease Drugs Uptake

This section focuses on the uptake rate of the potential Fabry Disease drugs recently launched in the Fabry Disease market or expected to be launched in 2020-2034. The analysis covers the Fabry Disease market uptake by drugs, patient uptake by therapies, and sales of each drug.

Fabry Disease Drugs Uptake helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on Fabry Disease market share and size, which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Fabry Disease Pipeline Development Activities

The Fabry Disease report provides insights into different therapeutic candidates in Phase II, and Phase III stages. It also analyses Fabry Disease key players involved in developing targeted therapeutics.

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Fabry Disease Pipeline Therapies and Key Companies

• PRX-102 (Pegunigalsidase Alfa): Protalix Biotherapeutics
• Venglustat: Sanofi Genzyme
• ST-920: Sangamo Therapeutics
• FLT190: Freeline Therapeutics
• 4D-310: 4D Molecular Therapeutics
• Lucerastat: Idorsia Pharmaceuticals
• Moss-aGal: GREENOVATION BIOTECH GMBH

Fabry Disease Therapeutics Assessment

Major key companies are working proactively in the Fabry Disease Therapeutics market to develop novel therapies which will drive the Fabry Disease treatment markets in the upcoming years are Protalix Biotherapeutics (NYSE: PLX), Sanofi Genzyme (EPA: SAN), Sangamo Therapeutics (NASDAQ: SGMO), Freeline Therapeutics (NASDAQ: FRLN), 4D Molecular Therapeutics (NASDAQ: FDMT), Idorsia Pharmaceuticals (SWX: IDIA), GREENOVATION BIOTECH GMBH, Shire (formerly NASDAQ: SHPG), Takeda (TSE: 4502), Amicus Therapeutics (NASDAQ: FOLD), uniQure (NASDAQ: QURE), Codexis (NASDAQ: CDXS), MP6 Therapeutics, CellGenTech, Protalix (NYSE: PLX) and others.

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Fabry Disease Report Key Insights

1. Fabry Disease Patient Population

2. Fabry Disease Market Size and Trends

3. Key Cross Competition in the Fabry Disease Market

4. Fabry Disease Market Dynamics (Key Drivers and Barriers)

5. Fabry Disease Market Opportunities

6. Fabry Disease Therapeutic Approaches

7. Fabry Disease Pipeline Analysis

8. Fabry Disease Current Treatment Practices/Algorithm

9. Impact of Emerging Therapies on the Fabry Disease Market

Table of Contents

1. Key Insights

2. Executive Summary

3. Fabry Disease Competitive Intelligence Analysis

4. Fabry Disease Market Overview at a Glance

5. Fabry Disease Disease Background and Overview

6. Fabry Disease Patient Journey

7. Fabry Disease Epidemiology and Patient Population

8. Fabry Disease Treatment Algorithm, Current Treatment, and Medical Practices

9. Fabry Disease Unmet Needs

10. Key Endpoints of Fabry Disease Treatment

11. Fabry Disease Marketed Products

12. Fabry Disease Emerging Therapies

13. Fabry Disease Seven Major Market Analysis

14. Attribute Analysis

15. Fabry Disease Market Outlook (7 major markets)

16. Fabry Disease Access and Reimbursement Overview

17. KOL Views on the Fabry Disease Market

18. Fabry Disease Market Drivers

19. Fabry Disease Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

About DelveInsight

DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.

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