Alpha Mannosidosis Market
Alpha Mannosidosis Market (Treatment - Bone Marrow Transplant (BMT) and Enzyme Replacement Therapy (ERT); Indication - Type I, Type II and Type III; End Users - Hospitals and Specialty Clinics) - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast 2019 - 2027
- According to Transparency Market Research’s latest report on the global alpha mannosidosis market for the historical period 2017–2018 and forecast period 2019–2027, market exclusivity for orphan drugs, increasing investment in rare disease treatment, and faster market access are projected to drive the global alpha mannosidosis market during the forecast period
- According to the report, the global alpha mannosidosis market was valued at US$ 7.6 Mn in 2018 and is anticipated to expand at a CAGR of 9.9% from 2019 to 2027
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Market Exclusivity for Orphan Drugs and Increasing Investment in Rare Disease Treatment: Key Drivers
- The most important financial incentive of orphan drug approval for pharmaceutical companies is the market exclusivity of several years for products that have been designated as orphan drugs.
- This allows pharmaceutical companies to recover costs incurred in development, as during this period regulatory agencies cannot approve a generic equivalent of the drug for the same rare indication.
- In the U.S., the period for market exclusivity for orphan drugs is 7 years and in the EU it is 10 years. Similar incentives exist in Japan and Australia, among other major regions
- Smaller clinical trial size, shorter trial time, and commercial benefits such as fast track approval, and fee waivers attract multinational corporations to invest in the rare disease segment
- The regulatory environment has been more advantageous for developers since the passage of the 1983 Orphan Drug Act. Moreover, venture capitalists see the legislative incentives as a major opportunity to create returns on their investments
- Moreover, saturation of the markets for diseases with large patient populations, such as asthma, diabetes and hypertension, has led to the focus of pharmaceutical industry towards more specialist, niche diseases where the unmet need is more substantial and the barriers to entry are much lower.
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Fee Reductions & Tax Credits Boost Market Growth
- Orphan drug designation qualifies the orphan drug manufacturer to claim tax credits of up to 50% of R&D costs incurred during the clinical trial phase in the U.S.
- According to a study conducted by the U.S. Congress, Office of Technology Assessment (OTA, 1993), overall the tax credit lowers the cost of the extra dollar of clinical orphan research by 24% compared to cost without tax credit
- Similar incentives include FDA fee waivers that reduces transaction cost associated with the approval process
- Therefore, fee reductions & tax credits is likely to increase demand for alpha mannosidosis globally
High Cost of Treatment to Hamper Market
- The most important barrier in this market is high cost of producing an orphan drug. The number of patients eligible to receive an orphan drug confines a company's ability to recover its R&D investment.
- Manufactures require incentives to develop orphan drugs. This being a narrow market, profit is based on supply and demand. By order of magnitude, these orphan drugs are more expensive.
- High investment involved in research and costs incurred for production of recombinant enzymes, increases cost of ERT. The per year treatment cost of a single lysosomal storage disorder patient is several hundred thousand dollars. Also, such high costs are acceptable in developed countries with established reimbursement structure however, in underdeveloped countries these are unaffordable and limits the access.
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Global Alpha mannosidosis Market: Competitive Landscape
- This report profiles major players in the global alpha mannosidosis market based on various attributes such as company overview, financial overview, product portfolio, business strategies, and recent developments
- The global alpha mannosidosis market is highly consolidated, with the presence of very few players
- The only player operating in the global alpha mannosidosis market is Zymenex A/S
Global Alpha mannosidosis Market: Key Developments
- CHIESI Farmaceutici S.p.A has adopted the business strategy of mergers and acquisitions. For instance, in 2013, CHIESI Farmaceutici S.p.A acquired the Zymenex A/S and entered into the treatment of rare disease market.
The report on the global alpha mannosidosis market discussed individual strategies, followed by company profile of manufacturer of alpha mannosidosis. The competitive landscape section has been included in the report to provide readers with a dashboard view and a company market share analysis of key players operating in the global alpha mannosidosis market.
Alpha Mannosidosis Market: Snapshot
Alpha mannosidosis is a rare genetic disorder, categorized as a lysosomal storage disorder. It is characterized by hearing loss, skeletal abnormalities, intellectual disability, coarse facial features, and ataxia. Some of the most common symptoms may include loss of hearing, distinctive facial features, mild to moderate intellectual disability, cerebellar disorders, and weakened immune system. Treatment solutions in global alpha mannosidosis market include various vaccinations, hearing aids, orthopedic assistance devices and equipment, speech therapy, antibiotics, glasses, and educational interventions. These products and services in global alpha mannosidosis market are only used when deemed necessary and professionals invite regular follow-ups to monitor patients’ health. Treatment solutions in global alpha mannosidosis market are based on the type of the disorder, which is classified into three types based on the severity of the symptoms. Type 1 is relatively mild, recognizable after the age of ten years, and displays a slow rate of progression. Type 2 is considered to be a moderate form, can be recognized before the age of ten, and includes myopathy and skeletal abnormalities. This is considered to be the most common forms of the disorder for treatment in global alpha mannosidosis market. Type 3 is considered to be severe, can manifest as loss in pregnancy or result in early death due to progressive central nervous system involvement.
Some of the most effective treatment options in global alpha mannosidosis market include peripheral blood stem cell transplantation and bone marrow transplant. Bone marrow transplant (BMT) is traditionally preferred method of treatment in global alpha mannosidosis market. Methods such as these are considered expensive as they rely on super specialty biomaterials and equipment to effectively administer. One of the emerging therapy solutions in global alpha mannosidosis market is enzyme replacement therapy. Increasing investments in research and development activities to promote discovering treatments for rare diseases is anticipated to propel the growth of global alpha mannosidosis market in coming years. However, high cost of treatments and lack of awareness regarding the alpha mannosidosis disorder can restrict the growth of global alpha mannosidosis market in near future.
TMR’s report on the global alpha mannosidosis market studies past as well as current growth trends and opportunities to gain valuable insights of these indicators of the market during the forecast period from 2019 to 2027. The report provides revenue of the global alpha mannosidosis market for the period 2017–2027, considering 2018 as the base year and 2027 as the forecast year. The report also provides the compound annual growth rate (CAGR) for the global alpha mannosidosis market during the forecast period.
The report has been prepared after an extensive primary and secondary research. Primary research involved bulk of research efforts, wherein analysts carried out interviews with industry leaders and opinion makers. Extensive secondary research involved referring to key players’ product literature, annual reports, press releases, and relevant documents to understand the global alpha mannosidosis market.
Secondary research also included Internet sources, statistical data from government agencies, websites, company presentations, sales data, and trade associations. Analysts have employed a combination of top-down and bottom-up approaches to study various phenomenon in the global alpha mannosidosis market.
The report includes an elaborate executive summary, along with a snapshot of the growth behavior of various segments included in the scope of the study. Furthermore, the report sheds light on the changing competitive dynamics in the global alpha mannosidosis market. These indices serve as valuable tools for existing market players as well as for entities interested in entering the global alpha mannosidosis market.
The report delves into the competitive landscape of the global alpha mannosidosis market. Key players operating in the global alpha mannosidosis market have been identified, and each one of these has been profiled for distinguishing business attributes. Company overview, financial standings, recent developments, and SWOT are some of the attributes of players in the global alpha mannosidosis market that have been profiled in this report.
Key Questions Answered in Global Alpha mannosidosis Market Report
- What is the scope of growth of product companies in the global alpha mannosidosis market?
- What will be the Y-o-Y growth of the global alpha mannosidosis market between 2019 and 2027?
- What is the influence of changing trends in technologies on the global alpha mannosidosis market?
- Will North America continue to be the most profitable market for alpha mannosidosis providers?
- Which factors are anticipated to hamper the growth of the global alpha mannosidosis market during the forecast period?
- Which are the leading companies in the global alpha mannosidosis market?
A unique research methodology has been utilized by TMR to conduct comprehensive research on the growth of the global alpha mannosidosis market and arrive at conclusions on its growth prospects. This research methodology is a combination of primary and secondary research, which helps analysts warrant the accuracy and reliability of the drawn conclusions.
Secondary methods referred to by analysts during the production of the global alpha mannosidosis market report include statistics from company annual reports, SEC filings, company websites, investor presentations, regulatory databases, government publications, and industry white papers. Analysts have also interviewed senior managers, product portfolio managers, CEOs, VPs, and market intelligence managers, who contributed to the production of TMR’s study on the alpha mannosidosis market as primary methods.
These primary and secondary methods have provided exclusive information during interviews, which serves as a validation from the alpha mannosidosis market leaders. Access to an extensive internal repository and external proprietary databases enabled this report to address specific details and questions about the global alpha mannosidosis market with accuracy. The study also uses the top-down approach to assess the revenues for each segment and the bottom-up approach to counter-validate them. This has helped in reaching TMR’s estimates on future prospects of the global alpha mannosidosis market more reliably and accurately.
Regional Segmentation of Alpha mannosidosis Market
- North America
- Rest of Europe
- Asia Pacific
- Australia & New Zealand
- Rest of Asia Pacific
- Latin America
- Rest of Latin America
- Middle East & Africa
- GCC Countries
- South Africa
- Rest of Middle East & Africa?
Global Alpha mannosidosis Market - Segmentation
Bone Marrow Transplant
Enzyme Replacement Therapy
Middle East & Africa