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Transthyretin (TTR) Amyloidosis Market | by Disease Type, Treatment, Gender, End User, and Region
Industry Research Report – Transthyretin (TTR) Amyloidosis Market forecast till 2023, is latest research report published by MRFR. The Global TTR Amyloidosis Market is segmented on the basis of treatment, gender, end-user, disease type, and Region.
Transthyretin (TTR) Amyloidosis Market Overview
The Transthyretin (TTR) amyloidosis market is likely to register a robust growth over the forecast period. Transthyretin (TTR) amyloidosis is a health condition characterized by anomalous deposits of a protein named amyloid (amyloidosis) in a person's tissues and organs. It usually affects autonomic neuropathy or peripheral neuropathic system and cardiac system. Amyloidosis symptoms are often ambiguous having similarity to symptoms caused by other conditions. The symptoms vary based on the location and type of the amyloid deposits. Primarily, three major types of transthyretin amyloidosis have been identified. The neuropathic type of transthyretin amyloidosis affects the autonomic and peripheral nervous systems, resulting in trouble controlling bodily functions and peripheral neuropathy. The leptomeningeal type of transthyretin amyloidosis mostly affects the central nervous system. The cardiac type of Transthyretin (TTR) amyloidosis puts patients at the risk of orthostatic hypertension, cardiomegaly, and arrhythmia.
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The surging African American population, increasing number of ATTR therapeutic drug launches, increasing average income of individuals, and growing healthcare awareness among people serves as key factors for driving the Transthyretin (TTR) amyloidosis market growth.
However, progress of the market can be restricted by strict governing policies, incorrect diagnosis of TTR ailments, restraints on clinical trials, and high cost of TTR drugs. Unavailability of advanced diagnostic methods in the middle-income nations and lack of knowledge about Transthyretin amyloidosis acts as major challenges to the market.
Transthyretin (TTR) Amyloidosis Market Segmentation
The worldwide Transthyretin (TTR) amyloidosis market has been segmented based on treatment, gender, end-user, and disease type. Based on treatment, the market has been segmented into organ transplantation, RNAi therapy, small molecules treatment, and others. Small molecules treatment has been sub-segmented as diflunisal and tafamidis. Based on gender, the market has been segmented into male and female. Based on end-user, the market has been segmented into ambulatory surgical centers, hospitals and clinics, and others. Based on disease type, the market has been segmented into Transthyretin (TTR) familial amyloid polyneuropathy, Transthyretin (TTR) familial amyloid cardiomyopathy, and others.
Transthyretin (TTR) Amyloidosis Market Regional Analysis
The Americas hold the largest market share for Transthyretin (TTR) amyloidosis. The growth is owing to the rising occurrence of amyloidosis among adults and older population and increasing elderly population. For instance, a report published by ASCO Journal in 2016 stated that almost 4,000 people suffered from amyloidosis each year in the United States. Additionally, increasing government funding, technologically advanced diagnostic equipment, and surging research initiatives in advanced medical treatment options are likely to drive the market. The existence of leading pharmaceutical brands such as Merck and Pfizer also boost the market in this region.
Europe represents the second largest share in the global Transthyretin (TTR) amyloidosis market. The Europe market has been anticipated to grow remarkably over the forecast period. The growth is owing to soaring healthcare expenditure, availability of progressive treatment facilities, amplifying demand for improved healthcare infrastructure collectively with government initiatives for healthcare reform. In the U.K., almost 60 new cases of Transthyretin (TTR) amyloidosis are reported every year. Furthermore, increased affordability coupled with well-established medical device manufacturers accelerate the market to a great extent. However, increasing incidences of wrong diagnosis and the inefficiency of treatment procedures are likely to restrict the growth in the Europe market.
Asia Pacific market has been fast expanding owing to rising aged population. Moreover, increasing patient pool, government initiatives to enhance the healthcare facility, availability of favorable insurance reimbursement policies, and a rapid adoption of advanced technology are likely to stimulate the Transthyretin (TTR) amyloidosis market.
The Middle East and Africa market has been anticipated to register least growth owing to limited access to treatment facilities and lack of health awareness. In the Middle East, Saudi Arabia and the United Arab Emirates are the largest markets. The market growth has been attributed to the easy accessibility of specialty care centers and growth of healthcare industry. However, the market is expected to witness steady development due to the genetical predisposition of Africans for transthyretin amyloidosis and administrative initiatives to excel the quality of healthcare in the region.
Transthyretin (TTR) Amyloidosis Market Key Players
The global Transthyretin (TTR) amyloidosis market has been dominated by key players such as Pfizer, Alnylam Pharmaceuticals, Ionis Pharmaceuticals Inc., Arcturus Therapeutics, Corino Therapeutics Inc, and Proclara Bioscience.
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#transthyretin amyloidosis market#TTR amyloidosis market#transthyretin amyloidosis market size#transthyretin amyloidosis market share#TTR amyloidosis market CAGR#ATTR#TTR familial amyloid polyneuropathy#TTR familial amyloid cardiomyopathy
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Global Transthyretin Amyloidosis Treatment Market–Analysis and Demand with Forecast Overview to 2029
Published Date:December 2020
Robust pipeline of transthyretin amyloidosis treatment drugs is one of the major factors that is expected to propel growth of the market over the forecast period.
The global transthyretin amyloidosis treatment market accounted for US$ 40.5 million in 2019 and is anticipated to register a CAGR of 58.7%.
The report "Global Transthyretin Amyloidosis Treatment Market, By Drug (Inostersen, Partisiran, Tafamidis, and Others), By Indication (Wild Type ATTR Amyloidosis, and Hereditary ATTR Amyloidosis), By Distribution Channel (Retail Pharmacy, Hospital Pharmacy, and Online Pharmacy), and By Region (North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa) - Trends, Analysis and Forecast till 2029”.
Key Highlights:
In August 2018, Alnylam Pharmaceuticals, Inc. received the U.S. Food and Drug Administration (FDA) approval for its ONPATTRO (patisiran) lipid complex injection— a RNA interference (RNAi) therapeutic— indicated for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
In July 2018, Ionis Pharmaceuticals, Inc. and Akcea Therapeutics, Inc. received marketing authorization approval for its drug TEGSEDI (inotersen) from the European Commission (EC) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis.
Analyst View:
Increasing prevalence of transthyretin amyloidosis
The global transthyretin amyloidosis treatment market is likely to be driven by an increase in the prevalence of disease, rise in the population with African origin, increase in awareness, improvement in diagnostic procedures, improving health care services, rapid economic growth in developing countries, and rise in research and development activity. For instance, According to the Amyloidosis Foundation, there are roughly 126 different genetic variations in ATTR, with up to 53 types of genetic variations in non-transthyretin hereditary amyloidosis diseases. According to reports published on transthyretin amyloidosis, it has been estimated that nearly 10,000, or 1.1 per 100,000 individuals in the world are living with TTR-FAP. The age group of patients suffering from the disorder is between 30 and 40 years of age. It has also been observed that TTR-CM tends to affect older males aged 65 years and above. Familial amyloid polyneuropathy (TTR-FAP) leads to 100 different types of mutations in the transthyretin gene, which leads to protein misfolding. There is only a 50% chance of transferring the mutation to the next generation from an affected parent. On the other hand, the mutation that leads to familial amyloid cardiomyopathy is generally found in individuals of African origin. Amyloidosis related to age primarily affects Caucasian men who are aged 65 years and above.
Increasing healthcare awareness among people
The global ATTR market is likely to exhibit a robust growth during the forecast period due to the probability of rising number of ATTR therapeutic drugs launches, increasing African-American population as they are genetically susceptible to amyloidosis, increasing healthcare awareness among people, and rising average income of individuals. However, the growth of this market can be hindered by stringent regulatory policies, the high cost of ATTR drugs, and incorrect diagnosis of ATTR disorders and limitations of clinical trials. Lack of knowledge about this condition and unavailability of advanced diagnostic methods in the middle-income countries are the major challenges to the market.
Browse 60 market data tables* and 35 figures* through 140 slides and in-depth TOC on “Global Transthyretin Amyloidosis Treatment Market”, By Drug (Inostersen, Partisiran, Tafamidis, and Others), By Indication (Wild Type ATTR Amyloidosis, and Hereditary ATTR Amyloidosis), By Distribution Channel (Retail Pharmacy, Hospital Pharmacy, and Online Pharmacy), and By Region (North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa) - Trends, Analysis and Forecast till 2029
Key Market Insights from the report:
The global transthyretin amyloidosis treatment market accounted for US$ 40.5 million in 2019 and is anticipated to register a CAGR of 58.7%. The market report has been segmented on the basis of drug, indication, distribution channel, and region.
Depending upon indication, the hereditary ATTR amyloidosis segment is projected to grow at highest CAGR over the forecast period. The hATTR is caused by the misfolding of protein monomers resulting from the TTR.
Depending upon distribution channel, the hospital pharmacy segment is projected to grow at highest CAGR over the forecast period
By region, North Americas dominates the market for Transthyretin (TTR) amyloidosis owing to the increasing prevalence of amyloidosis among adults and older population and rising geriatric population which is a major trigger factor for this disease. The presence of pharmaceutical giants such as Pfizer, Merck, etc. also propels the market growth in this region.
#Global Transthyretin Amyloidosis Treatment Market Size#Global Transthyretin Amyloidosis Treatment Market Share#Global Transthyretin Amyloidosis Treatment Market Insights#Global Transthyretin Amyloidosis Treatment Market Outlook#Global Transthyretin Amyloidosis Treatment Market Forecast
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Transthyretin Amyloidosis Treatment Market Slated US$ 1,077 Mn by 2028| Prothena Corporation, Pfizer, Ionis Pharmaceuticals, Valeant, Celgene, Takeda, Johnson and Johnson, AstraZeneca, GlaxoSmithKline
Transthyretin Amyloidosis Treatment Market expecting for US$ 1,077.7 million, anticipated to register a CAGR of +58% by the term plan of 2021-28.
TTR amyloidosis is a specific type of amyloidosis that is very rare. TTR stands for transthyretin, a protein that is primarily made in the liver. TTR amyloidosis occurs when this protein “misfolds” or changes its shape in an abnormal way, and forms into fibrous clumps.
TTR amyloidosis is a systemic disease in which amyloid deposits can be visualized in most tissues such as skin, fat pad, rectal mu- cosa, gastric mucosa, nerve tissue or, myocardium. Tissue biopsy should be performed, ideally of an affected organ.
Liver transplantation removes the source of mutated TTR molecules and prolongs survival, with a 20-year survival of 55%.
On average, people with familial ATTR amyloidosis live for 7 to 12 years after they get their diagnosis, according to the Genetic and Rare Diseases Information Center. A study published in the journal Circulation found that people with wild-type ATTR amyloidosis live an average of about 4 years after diagnosis.
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Major Players Covered in this Report:
Prothena Corporation plc., Pfizer Inc., Ionis Pharmaceuticals Inc., Valeant Pharmaceuticals International Inc., Celgene Corporation, Takeda Pharmaceutical Company Ltd., Johnson and Johnson Pvt. Ltd., GlaxoSmithKline plc, Alnylam Pharmaceuticals Inc., SOM Innovation Biotech S.L., AstraZeneca plc.
Report Consultant announced latest research on growth factors and development of Global Transthyretin Amyloidosis Treatment Market. A detailed study accumulated to offer latest insights about acute features of the Transthyretin Amyloidosis Treatment market. The report contains different market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market. It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary and SWOT analysis.
Transthyretin Amyloidosis Treatment Market Study assures you to advise higher than your competition. With Structured tables and figures examining the Transthyretin Amyloidosis Treatment, the research document provides you a leading product, submarkets, revenue size and forecast to 2028.
The study report offers a comprehensive analysis of Transthyretin Amyloidosis Treatment market size across the globe as regional and country level market size analysis, CAGR estimation of market growth during the forecast period, revenue, key drivers, competitive background and sales analysis of the payers. Along with that, the report explains the major challenges and risks to face in the forecast period.
Market segmentation by drug:
Inostersen
Partisiran
Tafamidis
Others
Market segmentation by Indication:
Wild Type ATTR Amyloidosis
Hereditary ATTR Amyloidosis
Market segmentation by Distribution Channel:
Retail Pharmacy
Hospital Pharmacy
Online Pharmacy
Market segmentation by regions:
North America
Europe
Asia-Pacific
Middle East and Africa
Rest of the world
The research report of the Transthyretin Amyloidosis Treatment market offers broad analysis about the industry on the basis of different key segments. Moreover, the research report presents a comprehensive analysis about the opportunities, new products, and technological innovations in the market for the players.
Additionally, the research report on Transthyretin Amyloidosis Treatment market provides an in depth analysis about market status, market size, revenue share, industry development trends, products’ advantages and disadvantages of the enterprise, enterprise competition pattern, industrial policy and regional industrial layout characteristics. Thus the study report offers a comprehensive analysis of market size across the globe as regional and country level market size analysis, estimation of market growth during the forecast period.
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This study also covers company profiling, specifications and product picture, sales, market share and contact information of various regional, international and local vendors of Global Transthyretin Amyloidosis Treatment Market. The market opposition is frequently developing greater with the rise in scientific innovation and M&A activities in the industry. Additionally, many local and regional vendors are offering specific application products for varied end-users. The new merchant applicants in the market are finding it hard to compete with the international vendors based on reliability, quality and modernism in technology.
Detailed TOC of Transthyretin Amyloidosis Treatment Market Research Report-
– Transthyretin Amyloidosis Treatment Introduction and Market Overview
– Transthyretin Amyloidosis Treatment Market, by Application
– Transthyretin Amyloidosis Treatment Industry Chain Analysis
– Transthyretin Amyloidosis Treatment Market, by Type
– Industry Manufacture, Consumption, Export, Import by Regions
– Industry Value ($) by Region
– Transthyretin Amyloidosis Treatment Market Status and SWOT Analysis by Regions
– Major Region of Transthyretin Amyloidosis Treatment Market
i) Global Transthyretin Amyloidosis Treatment Sales
ii) Global Transthyretin Amyloidosis Treatment Revenue & market share
– Major Companies List
– Conclusion
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Transthyretin Amyloidosis - Global Drug Forecast and Market Analysis to 2029 published on
https://www.sandlerresearch.org/transthyretin-amyloidosis-global-drug-forecast-and-market-analysis-to-2029.html
Transthyretin Amyloidosis - Global Drug Forecast and Market Analysis to 2029
Transthyretin Amyloidosis – Global Drug Forecast and Market Analysis to 2029
Summary
Amyloidosis is a protein confirmation disorder that results in the build-up of insoluble amyloid proteins in tissues. This build-up can be fatal as it leads to disruption of organ systems and causes oxidative stress within the body. There are various forms of amyloidosis, each associated with the protein involved and the underlying cause of protein mis-folding. Amyloid aggregates affect different organs, most commonly the heart, kidney and liver whilst also affecting the peripheral, autonomic and gastro-intestinal (GI) systems. Most amyloidosis diagnoses are made later in life, with the average patient aged 65 at diagnosis.
Transthyretin Amyloidosis (ATTR) is caused by the transthyretin (TTR) protein and occurs in two forms, either hereditary or wild type. Majority of organ involvement in ATTR is found in the heart and nerves are also affected. Liver transplant is a plausible treatment option for patients with ATTR-m as the liver is the cause of the mutated TTR protein, therefore removing the source of the issue seems to work well. However, a process described as seeding’ generally means that post-transplantation, the amyloid deposits eventually return due to the amyloid template left by pre-transplant mis-folding.
There are a number of unmet needs in the ATTR market, but drug treatment innovation is on the rise and diagnosis rates are expected to dramatically increase.
Key Highlights
– The main driver of the ATTR market expansion will be the approval and launch of the RNA interference therapeutics both ATTR polyneuropathy and ATTR cardiomyopathy. The approval of other pipeline agents of varying mechanism will also drive growth due to the current scarcity of the ATTR market. – Another prominent contributor to sales growth the increase in diagnosed prevalence across the 7MM. This increase will be caused by improved understanding and awareness of the disease alongside campaigns by drug developers to increase genetic testing. – The major global barrier for the ATTR market will be the annual cost of therapy for new pipeline drugs and the lack of options for advanced disease states, the stage at which majority of patients are diagnosed. – The key market opportunities lie in addressing unmet needs through the development of efficacious therapies for clearing already deposited amyloid and increasing the number of therapies approved for ATTR cardiomyopathy.
The report will enable you to – – Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Additionally a list of acquisition targets included in the pipeline product company list. – Develop business strategies by understanding the trends shaping and driving the global ATTR therapeutics market. – Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global ATTR therapeutics market in future. – Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors. – Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage. – Track drug sales in the global ATTR therapeutics market from 2019-2029. – Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.
Scope
– Overview of ATTR, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines. – Annualized ATTR therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2019 and forecast for ten years to 2029. – Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the ATTR therapeutics market. – Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs. – Analysis of the current and future market competition in the global ATTR market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.
Reasons to Buy
– A number of new therapies of varying mechanisms will enter the ATTR market over the forecast period. What strengths do these drugs bring to the market? – What are the main unmet needs in this market? Will the drugs under development fulfil the unmet needs of the ATTR market? – The current late-stage ATTR pipeline consists of a number of novel mechanisms such as the RNA interference therapies, Alnylam’s Onpattro, vutrisiran, and AKCEA’s Tegsedi and AKCEA-TTR-LRx, and Eidos’ TTR stabilizer, AG10. Will the late-stage drugs make a significant impact on the ATTR market? – Which of these drugs will have the highest peak sales at the highest CAGR, and why? – The ATTR patient population is currently very small, and the disease is said to hold a falsely perceived rarity. However, the diagnosed patient population is expected to dramatically increase over the forecast period. Why will this occur? – How will epidemiological changes impact the growth of the future market?
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Global Amyloidosis Treatment Market 2019 Worldwide Robust Expansion by Top Key Manufactures, Overview, Size, Share, Trends, Segments, Demand and Forecast to 2026
Global amyloidosis treatment market is expected to grow at a substantial CAGR in the forecast period of 2019-2026. Emergence of drugs used to treat risk associated with amyloidosis and high demand of disease specific novel therapies are the key factors that fueling the market growth.
Few of the major competitors currently working in the global amyloidosis treatment market are Takeda Pharmaceutical Company Ltd, Arcturus Therapeutics, Inc, Neurimmune, Pfizer Inc, Alnylam Pharmaceuticals, Inc, Proclara Biosciences, Fortress Biotech, Prothena, Spectrum Pharmaceuticals, Inc, Ionis Pharmaceuticals, Inc, Johnson & Johnson Services, Inc, Celgene Corporation, Eidos Therapeutics, GlaxoSmithKline plc, Amgen Inc, Fresenius Kabi AG, and others.
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Key Developments in the Market:
In June 2019, Alnylam Pharmaceuticals, Inc received an approval from the Japanese Ministry of Health, Labour and Welfare (MHLW) for Onpattro (patisiran), RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis with polyneuropathy. With this approval, represents major advances in the treatment of these patients through targeted mode of action.
In May 2019, Pfizer Inc, received an approval from the FDA for both Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis), an oral transthyretin stabilizers for the treatment of hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. The FDA approval of Vyndaqel and Vyndamax greatly enhance the company’s business portfolio in the therapeutic area of metabolic disorder.
Competitive Analysis:
Global amyloidosis treatment market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of global amyloidosis treatment market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.
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Transthyretin Amyloidosis Treatment Market Advanced Business Strategies & Competitive Dynamics
Hereditary transthyretin amyloidosis (hATTR) and wild type transthyretin amyloidosis are two main types of transthyretin amyloidosis. hATTR is further classified into Familial Amyloid Polyneuropathy (FAP) and Familial Amyloid Cardiomyopathy (FAC). Wild type variant (ATTRwt) predominantly affects the heart.
Transthyretin amyloidosis is a result of transthyretin produced by the liver and forms dimers, followed by monomers. Monomers aggregate to form amyloid fibrils, which are deposited in organs such as heart, nervous system, gastrointestinal tract, and kidneys. FAP is a subtype of hereditary transthyretin amyloidosis and the most common type of FAP is caused by the Val30Met variant of Transthyretin (TTR). In familial amyloid polyneuropathy, the symptoms are first detected after the patient crosses 30 years of age, however, it can also be detected as early as 20 years or as late as 80 years of age. Symptoms are divided depending on the location such as peripheral neuropathy and autonomic neuropathy. Symptoms may worsen in case excess amyloid protein starts to collect in the nerves.
Introduction of novel therapy for the treatment of transthyretin amyloidosis is expected to significantly drive the market growth
Key players in the market are focused on approval and launch of novel therapies for treatment of transthyretin amyloidosis. This in turn is expected to propel growth of the global transthyretin amyloidosis treatment market over the forecast period. For instance, in August 2018, Alnylam Pharmaceuticals, Inc. received the U.S. Food and Drug Administration (FDA) approval for its ONPATTRO (patisiran) lipid complex injection— a RNA interference (RNAi) therapeutic— indicated for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
In July 2018, Ionis Pharmaceuticals, Inc. and Akcea Therapeutics, Inc. received marketing authorization approval for its drug TEGSEDI (inotersen) from the European Commission (EC) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis.
The global transthyretin amyloidosis treatment market size is expected to be valued at US$ 35.8 million in 2018 and is expected to witness a robust CAGR of 55.4% over the forecast period (2018–2026).
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Major players in the market have novel drugs in the pipeline, which are in late-stage clinical trials and are expected to receive approval in the near future. For instance, Eidos Therapeutics, Inc. is developing AG10— an orally-administered, small molecule designed to potently and selectively stabilize tetrameric TTR, thereby interfering with events that give rise to ATTR. The drug is currently in Phase 2 clinical trial.
Akcea Therapeutics Inc. is co-developing AKCEA-TTR- LRx with Ionis Pharmaceuticals to inhibit the production of transthyretin. Akcea Therapeutics Inc. is focused on developing AKCEA-TTR-LRx for patients with both hereditary and wild type form of the disease. AKCEA-TTR-LRx is planning to enter clinical development in 2018. Key players in the market are focused on strategic partnerships in order to enhance their market share. For instance, in August 2018, Alnylam Pharmaceuticals, Inc. partnered with Orsini Healthcare, a specialty pharmacy as distributor of ONPATTRO (patisiran) lipid complex injection.
However, high costs of recently launched drugs is expected to hinder growth of the market. For instance, according to Alnylam Pharmaceutical, Inc., the cost of Patisiran is around US$ 450,000, annually before insurance. Another drug, Inotersen manufactured by Ionis Pharmaceuticals in partnership with Akcea Therapeutics, received approval for hereditary Transthyretin Amyloidosis Polyneuropathy indication and the drug costs US$ 450,000, annually. High cost of drugs may result in lower adoption even in developed economies such as the U.S., thereby restraining growth of the market.
Key players operating in the global transthyretin amyloidosis treatment market include, Alnylam Pharmaceuticals, Inc., Pfizer, Inc., Prothena Corporation Plc., GlaxoSmithKline Plc., Ionis Pharmaceuticals, Inc., Eidos Therapeutics, and SOM Innovation Biotech, S.L.
Approvals and launches of novel therapies for the treatment of transthyretin amyloidosis is expected to be a major factor driving the market growth over the forecast period. In October 2018, Eidos Therapeutics, Inc. received U.S. Food and Drug Administration (FDA) Orphan Drug Designation for its drug candidate AG10, indicated for the treatment of ATTR. In October 2018, Ionis Pharmaceuticals, Inc. and its Akcea Therapeutics Inc., received approval for its drug TEGSEDITM (inotersen injection) in Canada for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR)
Furthermore, increasing number under development drugs for the treatment of transthyretin amyloidosis are expected to gain approval in the near future. Therefore, presence of such robust pipeline is expected to boost the market growth. For instance, GlaxoSmithKline plc. a pharmaceutical company, is developing dezamizumab (2398852)† +2315698†, a combination of GSK2398852 (anti-SAP mAb) and GSK2315698 (CPHPC) for the treatment of amyloidosis. Currently, the product is under phase 2 clinical trial.
Transthyretin amyloidosis is a rare inherited and slowly progressive disease characterized by abnormal buildup of deposits of a protein called amyloid (amyloidosis) in the body's organs and tissues. Two main type of transthyretin amyloidosis includes hereditary transthyretin amyloidosis and wild type transthyretin amyloidosis. Hereditary transthyretin amyloidosis is further divided into polyneuropathy, cardiomyopathy, and mixed type. Diagnosis of transthyretin amyloidosis includes approaches such as imaging, histopathology, and molecular and genetic testing. Cardiac biomarkers such as N-terminal fragment of brain natriuretic peptide (NT-proBNP) and troponin are present in abnormally high concentrations in the heart, following amyloid deposits that can be tested for diagnosis of the disease. Genetic testing for transthyretin gene mutations is performed in case of familial transthyretin amyloidosis diagnosis.
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