The Farber Disease market report also offers comprehensive insights into the Farber Disease market size, share, Farber Disease epidemiology, emerging therapies, market drivers and barriers, ongoing clinical trials, key collaboration in the space, market uptake by key therapies and companies actively pushing Farber Disease market size growth forward.
Some of the key highlights from the Farber Disease Market Insights Report:
Several key pharmaceutical companies, including Hoffmann-La Roche Ltd, Novartis AG, AstraZeneca, Pfizer Inc., Sanofi, Johnson & Johnson Services Inc., AbbVie Inc., Allergan, Merck, Amgen, Daiichi Sankyo Company Ltd, Sun Pharmaceutical Industries Ltd., Teva Pharmaceutical Industries Ltd., Takeda Pharmaceutical Company Limited., GlaxoSmithKline, Cardinal Health, Mylan N.V., Boehringer Ingelheim International GmbH, Kowa Pharmaceuticals America, WOCKHARDT, and others, are developing novel products to improve the Farber Disease treatment outlook.
Farber disease, or Farber lipogranulomatosis, is a rare genetic disorder caused by a deficiency in the enzyme acid ceramidase, leading to ceramide accumulation in body tissues. It is inherited in an autosomal recessive manner, requiring mutations in both copies of the gene from each parent.
The United States holds the largest share of the Farber disease market, significantly surpassing the EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan.
Within the EU4 and the UK, Germany led the market in 2023 with a size of approximately USD 1.12 million, followed by Italy, while Spain had the smallest market share.
In Japan, the Farber disease market was valued at USD 0.88 million in 2023 and is expected to grow throughout the forecast period (2020–2034).
Aceragen is developing ACG-801, a recombinant human acid ceramidase (rhAC), as a potential enzyme replacement therapy for acid ceramidase deficiency, which causes Farber disease. Pre-clinical results have demonstrated promising safety and efficacy, but final validation will depend on the outcomes of ongoing Phase III clinical trials.
In 2023, about 90 cases of Farber disease were reported across the seven major markets (7MM), with the number expected to grow by 2034 due to improved diagnostics and awareness. In the U.S., 57% of cases were diagnosed in 2023, with 55% occurring in females. The classic and severe forms of Farber disease, which are more symptomatic and progress faster, accounted for approximately 65% of U.S. cases.
Currently, there are no approved therapies for Farber disease, and treatment is focused on symptom management using analgesics, corticosteroids, and plastic surgery. Allogeneic hematopoietic stem cell transplantation shows promise for patients with minimal neurological symptoms. The drug development pipeline remains limited, with RVT-801/ACG-801 by Aceragen being the most advanced candidate. The rarity of the disease poses challenges for clinical trial recruitment, slowing treatment advancements.
In 2023, the Farber disease market across the 7MM was valued at approximately USD 18.4 million, with the U.S. accounting for 70% of the market share.
As per DelveInsight analysis, the Farber Disease market is anticipated to witness growth at a considerable CAGR
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Farber Disease Overview
Farber disease, or Farber lipogranulomatosis, often appears in infancy or early childhood with a variety of symptoms. Common features include painful, swollen joints that can impair mobility, hoarseness caused by granulomas in the throat and larynx, and subcutaneous nodules—lumps beneath the skin. Additional symptoms may include skin rashes, respiratory difficulties, and gastrointestinal problems.
The condition is caused by a deficiency of the enzyme acid ceramidase, leading to ceramide accumulation in body tissues. It is inherited in an autosomal recessive manner, requiring mutations in both copies of the ASAH1 gene from each parent for the disease to develop.
Diagnosis involves clinical evaluation and laboratory testing. Enzyme assays are used to confirm acid ceramidase deficiency in blood or tissue samples, while genetic testing can identify mutations in the ASAH1 gene. Imaging and tissue biopsies may also be performed to assess granuloma formation and support the diagnosis.
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Farber Disease Epidemiology Insights
In 2023, the EU4 and the UK represented around 35% of all prevalent cases of Farber disease.
In the United States, only 57% of Farber disease cases were diagnosed in the same year, with approximately 55% of those cases occurring in females.
Farber Disease Epidemiology Segmentation
DelveInsight’s Farber Disease market report is prepared on the basis of epidemiology model. It offers comprehensive insights to the Farber Disease historical patient pools and forecasted Farber Disease patients. The report provides in-depth data of various subtypes and for the same epidemiology is segmented further. The Farber Disease Market report proffers epidemiological analysis for the study period 2020-34 in the 7MM segmented into:
Farber Disease Prevalence
Age-Specific Farber Disease Prevalence
Gender-Specific Farber Disease Prevalence
Diagnosed and Treatable Cases of Farber Disease
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Farber Disease Market Outlook
In 2023, the total market for Farber disease therapies across the seven major markets (7MM) was valued at USD 18.4 million, with the U.S. accounting for approximately 70% of the market. The U.S. market outperformed the EU4 (Germany, Spain, Italy, and France), the UK, and Japan. Market growth is anticipated due to increased awareness, improved diagnostic techniques, and the potential launch of new therapies, such as RVT-801/ACG-801 by Aceragen. Key drivers include advancements in research and development, progress in emerging treatments, and a growing understanding of the disease.
Farber Disease Emerging Drugs
RVT-801/ ACG-801: Aceragen
Farber Disease Key Companies
Hoffmann-La Roche Ltd
Novartis AG
AstraZeneca
Pfizer Inc.
Sanofi
Johnson & Johnson Services Inc.
AbbVie Inc.
Allergan
Merck
Amgen
Daiichi Sankyo Company Ltd
Sun Pharmaceutical Industries Ltd.
Teva Pharmaceutical Industries Ltd.
Takeda Pharmaceutical Company Limited.
GlaxoSmithKline
Cardinal Health
Mylan N.V.
Boehringer Ingelheim International GmbH
Kowa Pharmaceuticals America
WOCKHARDT
For more information, visit Farber Disease Market Analysis, Patient Pool, and Emerging Therapies
Scope of the Farber Disease Market Report:
11 Years Forecast
7MM Coverage
Descriptive overview of Farber Disease, causes, signs and symptoms, diagnosis, treatment
Comprehensive insight into Farber Disease epidemiology in the 7MM
Farber Disease marketed and emerging therapies
Farber Disease companies
Farber Disease market drivers and barriers
Table of Contents:
1 Farber Disease Market Key Comprehensive Insights
2 Farber Disease Market Report Introduction
3 Competitive Intelligence Analysis for Farber Disease
4 Farber Disease Market Analysis Overview at a Glance
5 Executive Summary of Farber Disease
6 Farber Disease Epidemiology and Market Methodology
7 Farber Disease Epidemiology and Patient Population
8 Farber Disease Patient Journey
9 Farber Disease Treatment Algorithm, Farber Disease Current Treatment, and Medical Practices
10 Key Endpoints in Farber Disease Clinical Trials
11 Farber Disease Marketed Therapies
12 Farber Disease Emerging Therapies
13 Farber Disease: 7 Major Market Analysis
14 Attribute analysis
15 Access and Reimbursement Overview of Farber Disease
16 Farber Disease Market Key Opinion Leaders Reviews
18 Farber Disease Market Drivers
19 Farber Disease Market Barriers
20 SWOT Analysis
21 Disclaimer
22 DelveInsight Capabilities
23 About DelveInsight
Related Reports:
Farber Disease Epidemiology 2034
DelveInsight’s “Farber Disease – Epidemiology Forecast to 2034” report delivers an in-depth understanding of the disease, historical and forecasted Farber Disease epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
Farber Disease Pipeline 2024
“Farber Disease Pipeline Insights, 2024” report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Farber Disease market. A detailed picture of the Farber Disease pipeline landscape is provided, which includes the disease overview and Farber Disease treatment guidelines.
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