Farbers Disease Market
DelveInsight’s ‘Farber’s disease – Market Insights, Epidemiology, and Market Forecast—2030’ report delivers an in-depth understanding of the Farber’s disease, historical and forecasted epidemiology as well as the market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
The Farber’s disease market report provides current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted Farber’s disease market Size from 2017 to 2030 segmented by seven major markets. The report also covers current Farber’s disease treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.
• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
Study Period: 2017–2030
Farber’s disease: Disease Understanding and Treatment Algorithm
Farber’s disease: Overview
Farber disease is also referred to as “Acid ceramidase deficiency,” “Farber lipogranulomatosis,” or “disseminated lipogranulomatosis.” It is an autosomal recessive lysosomal storage disease marked by a deficiency in enzyme ceramidase, which causes a progressive accumulation of fatty material lipids leading to abnormalities in the joints, liver, throat, tissues, and central nervous system.
The clinical presentation of Farber Disease (FD) is characterized by the appearance of subcutaneous skin nodules, ordinarily near the joints, most often interphalangeal, wrist, elbow, and ankle joints, or over points of mechanical pressure. These manifestations are very painful and lead to progressive joint stiffness, limitation of motion by contractures, and finally, immobilization and deformation of joints. Also, a characteristic sign of FD is the development of progressive hoarseness due to laryngeal involvement.
Besides these major manifestations, seven phenotypes have been described, which differ in severity and additional organ involvement, like the lungs, nervous system, heart, and lymph nodes. Dependent on residual lysosomal ceramidase turnover, patients have a variable degree of central nervous system disease, leading to progressive neurologic deterioration. In most cases, the neuronal dysfunction, rather than the general physical dystrophy, seems to limit the duration of FD. As well, patients with FD may die due to pulmonary disease with interstitial pneumonia.
Furthermore, researchers have described seven types of Farber lipogranulomatosis (type1, type2, type3, type4, type5, type6, type7) based on their characteristic features. The diagnosis is based on the clinical history and exam and specialized tests looking for white blood cell abnormalities. Genetic testing may also be used to confirm the diagnosis.
Farber’s disease is caused by a deficiency. Symptoms of the classic form may have moderately impaired mental ability and difficulty with swallowing. Other symptoms may include chronic shortening of muscles or tendons around joints, arthritis, swollen lymph nodes and joints, hoarseness, nodules under the skin (and sometimes in the lungs and other parts of the body), and vomiting. Some people may need a breathing tube. In severe cases, the liver and spleen are enlarged.
Farber’s disease Diagnosis and Treatment
It covers the details of conventional and current medical therapies and diagnosis available in the Farber’s disease market to treat the condition. It also provides the country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.
The DelveInsight Farber’s disease market report gives a thorough understanding of FARBER’S DISEASE symptoms by including details such as disease definition, symptoms, causes, pathophysiology, and diagnosis. It also provides the treatment algorithms and treatment guidelines for Farber’s disease symptoms in the US, Europe, and Japan.
Farber’s disease Epidemiology
The Farber’s disease symptoms epidemiology division provides insights about the historical and current patient pool along with the forecasted trend for every seven 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 DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.
The disease epidemiology covered in the report provides historical as well as forecasted Farber’s disease epidemiology segmented as the total diagnosed prevalent cases of Farber’s disease, gender-specific cases of Farber’s disease, and type-specific cases of Farber’s disease. The report includes the Prevalent scenario of Farber’s disease symptoms in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2017 to 2030.
Country-wise Farber’s disease Epidemiology
The epidemiology segment also provides the Farber’s disease epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
The total diagnosed prevalent population of Farber’s disease in the 7MM countries was estimated to be 86 cases in 2017.
Farber’s disease Drug Chapters
The drug chapter segment of the Farber’s disease report encloses the detailed analysis of Farber’s disease marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Farber’s disease clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.
There is no FDA-approved therapy for the treatment of Farber’s disease. The therapeutic market size of Farber’s disease in the US is mainly gauged by off-label treatment options such as Corticosteroids, Bone marrow transplants etc.
Products detail in the report…
Farber’s disease Emerging Drugs
RVT-801 (Enzyvant) is a recombinant form of human acid ceramidase (rhAC) being developed as a potential enzyme replacement therapy for acid ceramidase deficiency, manifesting as Farber disease. It is being evaluated in preclinical studies to enable a clinical trial in patients with Farber disease. The company plans to commence clinical trials by 2020 at eight sites across seven countries participating in the study. RVT-801 was granted Rare Pediatric Disease, Fast Track designations. The drug has also received Orphan Drug designations by the FDA and EMA.
Products detail in the report…
Farber’s disease Market Outlook
The Farber’s disease market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Farber’s disease market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers, and demand for better technology.
This segment gives a thorough detail of Farber’s 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’s, 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 market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
According to DelveInsight, Farber’s disease market in the 7MM is expected to change in the study period 2017–2030.
The management and diagnosis of FD have not advanced significantly in the past decade, but given recent advances in understanding the timing and potential pathogenesis of FD, it is hopeful that the next decade will bring early diagnostics and novel therapeutics.
This section includes a glimpse of the Farber’s disease market in the 7MM. The market size of Farber’s disease in the seven major markets was found to be USD 17.8 million in 2017.
The United States: Market Outlook
This section provides the total market size and market size by therapies of Farber’s disease in the United States.
The United States accounts for the highest market size of Farber’s disease in comparison to the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
Currently, there is no specific treatment for Farber’s disease. The treatment of Farber’s Disease includes symptomatic treatment includes Corticosteroids, Analgesics, Anti-inflammatory medications, and others. Hematopoietic stem cell transfer (HSCT) is another therapeutic option and has been demonstrated to substantially improve mobility and pain in several FD patients lacking CNS involvement.
Seizures may be treated with drugs that reduce the seizure occurrence (anti-epileptics). Some infants may need to undergo surgical removal of nodules that form in the airways or mouth cavity. Sometimes, a tracheostomy may be required. This is creating a surgical opening in the neck to gain access to the windpipe (trachea). A tube is placed into this opening to allow for breathing. Some affected children may need a gastrostomy tube, in which a small, thin tube is inserted into the stomach through a small cut in the abdomen to allow the passage of food and liquid.
Anti-inflammatory medications and physical therapy can reduce or improve mobility issues and pain associated with joint disease. Surgery may be necessary for the removal of nodules in the hand.
EU-5 Countries: Market Outlook
The total Farber’s disease market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.
As some kind of leukocyte dysregulation causes the inflammatory component of this disorder, allogeneic hematopoietic stem cell transplantation can restore a healthy immune system and provide a curative option in Farber’s Disease patients without neurological involvement. Previous stem cell transplantations in two children with severe neurological involvement had resulted in a disappointing outcome, as both patients died of progressive deterioration of their neurological status. Consequently, stem cell transplantation does not appear to abolish or even reduce the neurotoxic effects of the abundant ceramide storage in the brain.
The present experience with hematopoietic stem cell transplantation in Farber Diseases is still limited. HSCT may not be appropriate for patients with CNS involvement as ceramide neurotoxicity may not be reversible by stem cell transplantation.
Research using disease models has provided insights into the pathology as well as the role of ACDase in the development of these conditions. Recent studies have highlighted possible biomarkers for an effective diagnosis of ACDase deficiency. Ongoing work is being conducted to evaluate the use of recombinant human ACDase (rhACDase) for the treatment of FD. Finally, gene therapy strategies for the treatment of ACDase deficiency are actively being pursued.
Japan Market Outlook
The total market size and market size by therapies of Farber’s disease in Japan are also mentioned.
Farber’s disease Drugs Uptake
This section focuses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2017–2030. The analysis covers Farber’s disease market uptake by drugs, patient uptake by therapies, and sales of each drug.
This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size, which again will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions.
Farber’s disease Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase II and Phase III stage. It also analyses Farber’s disease key players involved in developing targeted therapeutics.
Enzyvant is expected to get launched in the US market by 20XX.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for Farber’s disease emerging therapies.
KOL – Views
To keep up with current market trends, we take KOLs and SME’s opinion working in Farber’s disease domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Farber’s disease market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.
Competitive Intelligence Analysis
We perform Competitive and Market Intelligence analysis of the Farber’s disease market by using various Competitive Intelligence tools: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.
Scope of the Report
• The report covers the descriptive overview of Farber’s disease, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies
• Comprehensive insight has been provided into the Farber’s disease epidemiology and treatment in the 7MM
• Additionally, an all-inclusive account of both the current and emerging therapies for Farber’s disease is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
• A detailed review of Farber’s disease market; historical and forecasted, is included in the report, covering drug outreach in the 7MM
• The report provides an edge while developing business strategies by understanding trends shaping and driving the global Farber’s disease market
• In the coming years, Farber’s disease market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
• The companies and academics are working to assess challenges and seek opportunities that could influence Farber’s disease R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
• Major players are involved in developing therapies for Farber’s disease. The launch of emerging therapies will significantly impact the Farber’s disease market
• A better understanding of disease pathogenesis will also contribute to developing novel therapeutics for Farber’s disease.
• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities
Farber’s disease Report Insights
• Patient Population
• Therapeutic Approaches
• Farber’s disease Pipeline Analysis
• Farber’s disease market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies
Farber’s disease Report Key Strengths
• 11-years Forecast
• 7MM Coverage
• Farber’s disease Epidemiology Segmentation
• Key Competitors
• Highly Analyzed Market
• Drugs Uptake
Farber’s disease Report Assessment
• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Market Drivers and Barriers
• What were the Farber’s disease Market share (%) distribution in 2017, and how would it look like in 2030?
• What would be the Farber’s disease total market Size as well as market Size by therapies across the 7MM during the forecast period (2017–2030)?
• What are the key findings pertaining to the market across 7MM, and which country will have the largest Farber’s disease market Size during the forecast period (2017–2030)?
• At what CAGR, the Farber’s disease market is expected to grow in the 7MM during the forecast period (2017–2030)?
• What would be the Farber’s disease market outlook across the 7MM during the forecast period (2017–2030)?
• What would be the Farber’s disease market growth till 2030, and what will be the resultant market Size in the year 2030?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
• What are the disease risk, burden, and unmet needs of the Farber’s disease?
• What is the historical Farber’s disease patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan?
• What would be the forecasted patient pool of Farber’s disease in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Farber’s disease?
• Out of all 7MM countries, which country would have the highest prevalence of Farber’s disease during the forecast period (2017–2030)?
• At what CAGR the population is expected to grow in the 7MM during the forecast period (2017–2030)?
Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:
• What are the current options for the treatment of Farber’s disease?
• What are the current treatment guidelines for the treatment of Farber’s disease in the USA, Europe, and Japan?
• What are the Farber’s disease marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
• How many companies are developing therapies for the treatment of Farber’s disease?
• How many therapies are developed by each company for the treatment of Farber’s disease?
• How many emerging therapies are in the mid-stage and late stages of development for the treatment of Farber’s disease?
• What are the key collaborations (Industry–Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Farber’s disease therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Farber’s disease and their status?
• What are the key designations that have been granted for the emerging therapies for Farber’s disease?
• What are the global historical and forecasted market for Farber’s disease?
Reasons to buy
• The report will help in developing business strategies by understanding trends shaping and driving the Farber’s disease market,
• To understand the future market competition in the Farber’s disease market and Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for Farber’s disease in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for Farber’s disease market.
• To understand the future market competition in the Farber’s disease market.