Farber's Disease Global Market Report 2025

The farber's disease market size has grown strongly in recent years. It will grow from $1.87 billion in 2024 to $1.98 billion in 2025 at a compound annual growth rate (CAGR) of 6%. The growth during the historic period can be credited to increasing awareness and diagnosis rates, greater adoption of genetic testing, a rise in research efforts focused on lysosomal storage disorders, the expansion of rare disease registries, and growing support from patient advocacy organizations.

The farber's disease market size is expected to see strong growth in the next few years. It will grow to $2.48 billion in 2029 at a compound annual growth rate (CAGR) of 5.7%. The growth in the forecast period can be attributed to the expanding pipeline of targeted therapies, increasing government incentives and orphan drug designations, broader access to specialized healthcare services, rising investments in rare disease-focused biotech startups, and the growing use of next-generation sequencing. Key trends expected during this period include progress in gene therapy technologies, the integration of artificial intelligence for diagnosing rare diseases, technology-enabled biomarker discovery, advancements in enzyme replacement therapies, and improvements in newborn screening programs.

The increasing prevalence of rare genetic disorders is anticipated to drive the growth of the Farber’s disease market in the coming years. Rare genetic disorders are inherited conditions caused by mutations in an individual’s DNA, affecting a very small portion of the population. This rise in prevalence is largely attributed to advancements in diagnostic technologies that allow for earlier and more accurate detection of conditions that were previously undiagnosed or misdiagnosed. Farber’s disease plays a significant role in addressing the rise in rare genetic disorders by acting as a model for the development of targeted therapies and supporting research in enzyme replacement treatments for ultra-rare conditions. For example, in November 2022, the Australian Government Department of Health and Aged Care reported that rare diseases - defined as affecting fewer than 5 in 10,000 people - impact around 2 million individuals, or 8% of the population, with over 7,000 known chronic or life-threatening conditions. Hence, the rising prevalence of rare genetic diseases is contributing to the expansion of the Farber’s disease market.

The rising investment in clinical trials is expected to drive the growth of the Farber’s disease market in the coming years. Clinical trials are research studies conducted in humans to assess the safety, effectiveness, and outcomes of medical interventions, including drugs, treatments, or medical devices. This growing investment is fueled by the increasing demand for innovative therapies, spurred by advancements in medical science, personalized medicine, and the pressing need to address unmet healthcare challenges. Farber’s disease underscores the importance of such investments by emphasizing the need for research focused on rare and underserved genetic disorders. For example, in October 2024, Citeline, a US-based provider of pharmaceutical intelligence solutions, reported that TrialTrove recorded 9,959 Phase I-III clinical trials set to begin in 2024 involving at least one drug - a 9.4% rise compared to 2023. Thus, the growing investment in clinical trials is contributing to the expansion of the Farber’s disease market.

In September 2022, Idera Pharmaceuticals, a biotechnology company based in the US, acquired Aceragen for an undisclosed sum. This acquisition was intended to strengthen Idera Pharmaceuticals’ position in the rare disease market by adding Aceragen’s pipeline to its portfolio, including ACG-801, a therapy in development for Farber’s disease. The move aims to address a significant unmet medical need and accelerate the advancement of enzyme replacement therapies. Aceragen Inc., also a US-based biopharmaceutical company, is actively developing ACG-801, an enzyme replacement therapy designed specifically to treat Farber disease, a rare lysosomal storage disorder.

Major players in the farber's disease market are Pfizer Inc., Merck & Co. Inc., Sanofi S.A., Takeda Pharmaceutical Company Limited, Mount Sinai Health System, Emory Healthcare, Kyowa Kirin Co. Ltd., BioMarin Pharmaceutical Inc., Children’s National Hospital, Medanta - The Medicity (Global Health Ltd.), Amicus Therapeutics Inc., JCR Pharmaceuticals Co. Ltd., Spark Therapeutics Inc., REGENXBIO Inc., Passage Bio Inc., Protalix BioTherapeutics Inc., Medicover Hospitals, Minoryx Therapeutics S.L., bluebird bio Inc., and Avrobio Inc.

North America was the largest region in the farber's disease market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in farber's disease report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the farber's disease market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Farber's disease is a rare inherited disorder resulting from a deficiency of the enzyme acid ceramidase, which causes fatty substances to build up in body tissues. It is passed down through an autosomal recessive pattern and usually appears during infancy. Common symptoms include joint deformities, subcutaneous nodules, and a gradual decline in neurological function. The disease progresses over time and can be life-threatening, necessitating coordinated care and symptom-focused treatment.

Farber's disease primarily exists in two forms: classical and non-classical. The classical form is the most common type and is marked by early-onset symptoms such as joint abnormalities, subcutaneous nodules, and progressive neurological deterioration. Available treatments include enzyme replacement therapy, gene therapy, symptomatic management, bone marrow transplantation, and supportive interventions for symptoms like joint issues, hearing loss, respiratory problems, and skin-related conditions. Key end-users include hospitals, specialized clinics, diagnostic facilities, and others.

The farber's disease market research report is one of a series of new reports that provides farber's disease market statistics, including the farber's disease industry global market size, regional shares, competitors with the farber's disease market share, detailed farber's disease market segments, market trends, and opportunities, and any further data you may need to thrive in the farber's disease industry. This farber's disease market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

The farber's disease market includes revenues earned by rehabilitation services, nutritional support services, and clinical trial access services. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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