Fabry Disease Drugs Market Analysis North America, Europe, Asia, Rest of World (ROW) - US, Germany, UK, China, Japan - Size and Forecast 2024-2028

Fabry Disease Drugs Market 2024-2028

The global fabry disease drugs market size is estimated to grow by USD 859.06 million, at a CAGR of 7.43% between 2023 and 2028. 

Key market players have been actively investing in the research and development (R&D) of drugs for the treatment of Fabry disease. Several drugs from companies such as Takeda Pharmaceutical Co. Ltd., Idorsia Pharmaceuticals Ltd., and Sanofi, among others, are experiencing different stages of clinical trials for use in the treatment of Fabry disease. For example, in March 2023, Takeda Pharmaceutical Co. Ltd., in partnership with Centogene, announced the extension of genetic testing for lysosomal storage disorders such as Fabry disease for one more year. Also, CHIESI Farmaceutici SpA announced that the FDA had approved PRX-102 (pegunigalsidase alfa) for the treatment of Fabry disease in May 2023. Thus, such developments, particularly focused on the treatment of Fabry disease, will drive market growth during the forecast period.

Technavio has segmented the market into Distribution Channel, Route Of Administration, and Geography 

• The distribution channel segment includes hospital pharmacies, retail pharmacies, and online pharmacies
• The route of administration segment includes intravenous route and oral route
• The geography segment includes key regions such as North America, Europe, Asia, and Rest of World (ROW)

It also includes an in-depth analysis of drivers, trends, and challenges. Our market analysis and report examines historical data from 2018-2022, besides analyzing the current market scenario.

What will be the size of the Fabry Disease Drugs Market During the Forecast Period?

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Fabry Disease Drugs Market Segmentation by Distribution Channel, Route Of Administration, and Geography Analysis

Distribution Channel Analysis 

Hospital pharmacies 

The market share growth by the hospital pharmacies segment will be significant during the forecast period. Hospital pharmacies supply the pharmaceutical drugs that are administered to individuals during hospital-based inpatient or outpatient treatment. Hospital pharmacies are the preferred distribution channel for Fabry disease drugs like injectables and oral medications. These pharmacies provide advice on exact drug dosages, interactions, and potential side effects, as well as monitor patient adherence to medication regimens. This can help improve patient outcomes.

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The hospital pharmacies segment was valued at USD 981.00 million in 2018. Additionally, hospital pharmacies also play a key role in clinical trials. Clinical trials are conducted to test the safety and efficacy of new drugs, and hospital pharmacies are often involved in the preparation and dispensing of medications used in these trials. By participating in clinical trials, hospital pharmacies contribute to the development of new drugs, including for rare diseases like Fabry disease. Thus, such factors make hospital pharmacies a critical channel, which drives the growth of the segment. This, in turn, is expected to drive market growth during the forecast period.

Route Of Administration Analysis

Intravenous route

Due to the availability of patient assistance programs and the benefits of direct administration of medication, the intravenous route segment is witnessing steady growth. Market players also provide various programs to buy medications that are administered intravenously.  The major factors associated with the growth of the intravenous route segment include the low cost of treatment and the convenience of infrequent dosing. Furthermore, the availability of intravenous route Fabry disease drugs offered by market vendors will contribute to the growth of the global fabry disease drugs market during the forecast period.

Oral route

Easy-to-administer benefits of oral drugs and the growing popularity of prescription-based drugs, such as oral capsules, are driving the growth of the oral route segment of the global fabry disease drugs market. Oral therapy with migalastat seems to be a convenient alternative to intravenous enzyme replacement therapy for patients who have Fabry disease with amenable mutations. However, the side effects of these oral drugs and the presence of stringent regulations have resulted in the rejection of Fabry disease oral drugs. Nevertheless, various ongoing clinical trials for oral drugs to treat Fabry disease and their easy-to-use benefits are expected to drive the growth of the segment, which, in turn, is anticipated to propel the market growth during the forecast period.

Regional Analysis

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North America is estimated to contribute 40% to the growth of the global market during the forecast period. Technavio’s analysts have elaborately explained the regional trends and drivers that will shape the market during the forecast period. North America's Fabry disease drugs market is poised for growth, fueled by factors like ongoing trials, increased rare disease diagnoses, favorable reimbursement schemes, high drug prices, key players' presence, and support from government and non-profit organizations. Recent FDA approvals, like pegunigalsidase alfa-iwi (ELFABRIO), highlight advancements. Patient assistance programs, addressing the high cost of Fabry disease drugs, are crucial, reducing non-compliance risks and boosting demand. Moreover, the Patient Protection and Affordable Care Act enhances healthcare access, covering pre-existing conditions. The region's rising obesity rates, linked to Fabry nephropathy, further drive drug adoption. Increasing rare disease prevalence, aided by advanced genetic profiling, is expected to drive the growth of the market in focus in North America during the forecast period.

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Key Fabry Disease Drugs Market Players 

Companies are implementing various strategies, such as strategic alliances, partnerships, mergers and acquisitions, geographical expansion, and product/service launches, to enhance their presence in the market. The report also includes detailed analyses of the competitive landscape of the market and information about 8 market companies, including:

4D Molecular Therapeutics Inc.: The company offers fabry disease drug such as 4D 310 for the treatment of fabry disease cardiomyopathy, which is the primary cause of death and not addressed by current therapies.

• Amicus Therapeutics Inc.
• CHIESI Farmaceutici SpA
• Freeline Therapeutics Holdings plc
• Idorsia Pharmaceuticals Ltd.
• Sangamo Therapeutics Inc.
• Sanofi SA
• Takeda Pharmaceutical Co. Ltd.

Qualitative and quantitative analysis of companies has been conducted to help clients understand the wider business environment as well as the strengths and weaknesses of key market players. Data is qualitatively analyzed to categorize companies as pure play, category-focused, industry-focused, and diversified; it is quantitatively analyzed to categorize companies as dominant, leading, strong, tentative, and weak.

Fabry Disease Drugs Market: Key Drivers, Trends, Challenges, and Customer Landscape

There are multiple factors influencing market growth analysis. Our researchers analyzed the data with 2023 as the base year, along with the key drivers, trends, and challenges. 

Key Fabry Disease Drugs Market Drivers

The rising incidence of rare diseases is the key factor driving market growth. Fabry disease drugs are particularly used to treat a rare disease, Fabry disease. Most rare diseases contain inherited genetic disorders and neurodegenerative diseases. Paradoxically, though rare diseases are low in incidence collectively, they affect a significant proportion of the population in any country. Individual diseases may be rare, but the total population with a rare disease is large. According to the NIH, in the US, 1 out of 2 patients diagnosed with a rare disease is a child. There are different types of inherited genetic disorders, such as X-linked inheritance (like Fabry disease).

However, no diagnostic methods exist for many inherited rare diseases. 80% of all rare disease patients are affected by approximately 350 rare diseases, according to the Indian Health Ministry. Consequently, many diagnostic and pharmaceutical companies are studying rare diseases extensively. Thus, the rising number of people diagnosed with rare diseases like Fabry disease will increase the need for fabry disease drugs, which is expected to drive market growth during the forecast period. 

Significant Fabry Disease Drugs Market Trends

The growing number of awareness programs for Fabry disease is the primary trend shaping market growth. Fabry disease awareness is actively promoted globally through initiatives by market players and advocacy groups. In the US, the National Fabry Disease Foundation observes Fabry Disease Awareness Month annually in April. Eurordis, representing rare disease organizations in Europe, conducts awareness programs, including those for Fabry disease. The Fabry International Network designates April as "Fabry month" for awareness campaigns.

Moreover, key market player Sanofi, through its Rare Humanitarian program, raises awareness for rare diseases, including Fabry disease. Collaborating with the American Kidney Fund, Sanofi Genzyme launched an education and awareness campaign in September 2020 to address the unmet needs of the Fabry disease community. These global campaigns are anticipated to contribute to the growth of the fabry disease drugs market, aligning with market trends and analysis during the forecast period.

Major Fabry Disease Drugs Market Challenges

Loss of patents and patent expiry is a challenge that affects market growth.  The loss or expiry of patents eventually leads to an influx of generic products in the market, resulting in large players losing their competitive advantages and profit margins. Generic drugs are those drugs that are similar to branded drugs in terms of quality, dosage form, strength, and efficacy but are available at lower prices. The penetration of generic drugs limits market growth as the drugs become available at lower prices.

Further, allegations associated with the infringement of patents and other intellectual property rights of third parties affect the business of a company. The expiry of patents on branded Fabry disease drugs will result in the introduction of some generic versions of these drugs, thereby lowering the sales of branded drugs, which can negatively affect the business of prominent market players. Hence, the loss or expiry of patents on Fabry disease drugs impedes market growth during the forecast period.

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Key Fabry Disease Drugs Market Customer Landscape

The market report includes the adoption lifecycle of the market research and growth, covering from the innovator’s stage to the laggard’s stage. It focuses on adoption rates in different regions based on penetration. Furthermore, the report also includes key purchase criteria and drivers of price sensitivity to help companies evaluate and develop their growth strategies.

Global Fabry Disease Drugs Market Customer Landscape

Segment Overview

The fabry disease drugs market report forecasts market growth and forecasting by revenue at global, regional & country levels and provides an analysis of the latest market growth and trends from 2018 to 2028. 

• Distribution Channel Outlook
  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies

• Route Of Administration Outlook
  • Intravenous route
  • Oral route

• Region Outlook
  • North America
    • The U.S.
    • Canada
  • Europe
    • U.K.
    • Germany
    • France
    • Rest of Europe
  • Asia
    • China
    • India
  • Rest of World (ROW)
    • Brazil
    • Argentina
    • Australia

Fabry Disease Drugs Market Scope
Report Coverage	Details
Page number	149
Base year	2023
Historic period	2018 - 2022
Forecast period	2024-2028
Growth momentum & CAGR	Accelerate at a CAGR of 7.43%
Market growth 2024-2028	USD 859.06 million
Market structure	Concentrated
YoY growth 2023-2024(%)	6.83
Regional analysis	North America, Europe, Asia, and Rest of World (ROW)
Performing market contribution	North America at 40%
Key countries	US, Germany, UK, China, and Japan
Competitive landscape	Leading Vendors, Market Positioning of Vendors, Competitive Strategies, and Industry Risks
Key companies profiled	4D Molecular Therapeutics Inc., Amicus Therapeutics Inc., CHIESI Farmaceutici SpA, Freeline Therapeutics Holdings plc, Idorsia Pharmaceuticals Ltd., Sangamo Therapeutics Inc., Sanofi SA, and Takeda Pharmaceutical Co. Ltd.
Market dynamics	Parent market analysis, Market growth inducers and obstacles, Fast-growing and slow-growing segment analysis, COVID-19 impact and recovery analysis and future consumer dynamics, and market condition analysis for the forecast period.
Customization purview	If our report has not included the data that you are looking for, you can reach out to our analysts and get segments customized.

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What are the Key Data Covered in this Fabry Disease Drugs Market Research Report?

• CAGR of the market during the forecast period
• Detailed information on factors that will drive the growth of the fabry disease drugs market forecast between 2024 and 2028
• Precise estimation of the fabry disease drugs market size and its contribution to the market in focus on the parent market
• Detailed analysis of customer landscape covering Diver of price sensitivity, Adoption of the lifecycle, and key purchase criteria 
• Companies landscape comparing criticality of inputs and factors of differentiation
• Exclusive Matrix on companies' position and classification
• Accurate predictions about upcoming trends and changes in consumer behavior
• Growth of the market across North America, Europe, Asia, and Rest of World (ROW)
• A thorough analysis of the market’s competitive landscape and detailed information about companies
• Comprehensive analysis of factors that will challenge the growth of fabry disease drugs market companies

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