#Hereditary Amyloidosis Market Trend

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https://www.timessquarereporter.com/health/hereditary-amyloidosis-market-is-estimated-to-witness-high-growth-owing-to-increasing-prevalence-of-amyloidosis

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Hereditary Transthyretin Amyloidosis (hATTR) is a rare, progressive, and life-threatening genetic disorder caused by mutations in the transthyretin (TTR) gene.

[hustler0101]

US Hereditary Transthyretin Amyloidosis (HATTR)This report studies the Hereditary Transthyretin Amyloidosis (HATTR) market size (value and volume) by players, regions, product types and end industries, history data 2018-2022 and forecast data 2023-2030; This report also studies the global market competition landscape, market drivers and trends, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

[apurvasstuff]

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.

[fortunatelyfuturisticmentality]

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

[jesse-pinkman123]

Familial Amyloid Cardiomyopathy Treatment Market Opportunity Analysis-2026

Regional growth engines of the familial amyloid cardiomyopathy treatment

On the basis of regional segmentation, the Familial Amyloid Cardiomyopathy Market can be segmented into North America, Europe, Asia Pacific, Latin America, Middle East, and Africa. According to BioMed Central journal’s published data in year March 2017, Europeans had highest number of TTR mutations, followed by East Asians, American, Central-South Asians, and Africans which favors the growth of familial amyloid cardiomyopathy market.

Aggregation and deposition of mutant and wild-type transthyretin protein (TTR) in heart results in familial amyloid cardiomyopathy, which typically occur after age of 60. Familial amyloid cardiomyopathy is also known as hereditary cardiac transthyretin amyloidosis or hereditary amyloid cardiomyopathy. The protein transthyretin amyloid fibrils infiltrate the myocardium that leads to diastolic dysfunction from restrictive cardiomyopathy, which eventually result to heart failure. There are several mutation in TTR which are associated with familial amyloid cardiomyopathy, which includes V1221, P24S, V20I, A45T, Gly47Val, Glu51Gly, Gln92Lys, I68L, and L111M. Most common mutation is V1221, which is substitution of isoleucine for valine at position 122 that occurs in high frequency in African-Americans, which favors the growth of familial amyloid cardiomyopathy treatment market.

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European Medicines Agency have approved drug named tafamids (Vyndaqel) for slow progression of familial amyloid cardiomyopathy in year 2013 which is expected to favor the market of familial amyloid cardiomyopathy treatment. Most of the pharmaceuticals companies are developing drugs for familial amyloid cardiomyopathy treatment, as it is associated with progressive heart failure and it is universally fatal. Symptoms for familial amyloid cardiomyopathy are weight loss, nausea, fatigue, dizziness and collapse, palpitations, abnormal heart rhythms, disrupted sleep, and angina. Genetic tests are recommended for detection of TTR gene.

Rise in amount of transthyretin protein in the body fuels the familial amyloid cardiomyopathy treatment market

Transthyretin protein is formed in the body abundantly due to inherited transthyretin gene alteration, which leads to familial amyloid cardiomyopathy as gene associated transcribes the protein. Gene alteration in body leads to alteration in sequence of body proteins. Familial amyloid cardiomyopathy are inherited by autosomal dominant allele, which can inherit the familial amyloid cardiomyopathy condition to offsprings from one of the parent. Only one copy of mutated gene is sufficient to cause the disease, which is expected to show growth in the familial amyloid cardiomyopathy market. Familial amyloid cardiomyopathy is critical disease leading to heart failure, which can favor the market. According to Amyloidosis Foundation (AF), which provides early diagnosis and advanced treatment, familial amyloid cardiomyopathy are rarely found with statistics estimating 1 per 100,000 population suffering from familial amyloid cardiomyopathy disease.

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The key players dominating the familial amyloid cardiomyopathy market

Familial amyloid cardiomyopathy not only affect lives of people but also affect upcoming generations, due to which manufactures are in process of releasing the drugs, as there are very few drugs approved for familial amyloid cardiomyopathy treatment. Recently in June 2017 the U.S. Food and Drug Administration (FDA), which provides accurate, science-based health information to the public, granted fast track designation to tafamidis the company’s investigational treatment for familial amyloid cardiomyopathy as there were no approved treatment in the U.S. tafamidis with trade name VYNDAQEL® was first approved in Europe. The key players operating the market are Pfizer Inc., SOM Innovation Biotech, S.L., Valeant Pharmaceuticals International, Inc., Ionis Pharmaceuticals, Inc., and AstraZeneca plc.

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[sapanas]

Cardiomyopathy Medication Market 2021: Gross Margin Analysis, Global Overview, Emerging Trends, Leading Growth Drivers, Future Estimation

Cardiomyopathy Medication Market Overview

The global cardiomyopathy medication market size is foreseen to reach USD 620 Million by 2025 at a CAGR of 4.60% during the gauge time of 2019 to 2025. Cardiomyopathy is an infection that causes the thickening and growth of the heart muscles and strange blood stream. Patients experiencing cardiomyopathy feature manifestations, for example, wooziness, tiredness, and expansion to legs, lower legs, and feet.

Widened adoption of cardiomyopathy medication is a cardiovascular issue brought about by development of heart. The strong dividers of the heart become frail because of expansion and consequently heart can't siphon blood productively. The turmoil causes impact on liver, lungs and other significant organs of the body consistently. It is the clinical condition which causes augmentation of at first left vertical of the heart, which is considered as principle siphoning chamber. Altogether, if may spread to right chamber over the time. cardiomyopathy medication additionally prompts visit blood clumps, sporadic pulses and valve issues, which may influence any individual regardless of age and sex, although the likelihood proportion of male over female is 3:1.

Different medicines accessible for cardiomyopathy medication centers in progress of blood stream which is it fundamental cause. The treatment of the cardiomyopathy medication should be possible with two procedures one is by oral medication and other is implantable gadgets. The kinds of cardiomyopathy medication drugs accessible are beta blockers, diuretics, digitalis, angiotensin 2 receptor blocker, warfarin, diuretics and angiotensin-changing over chemical inhibitors. In the second classification of treatment, implantable gadgets, heart siphons and cardioverter-defibrillators are unmistakably utilized.

The primary driver of cardiomyopathy medication are diseases in cardiovascular muscles, hereditary qualities or birth deformities, medication or liquor misuse or certain presentation to lethal substances, for example, mercury, lead or cobalt. In a portion of the cases cardiomyopathy medication may likewise be caused as symptom of disease medication.

Cardiomyopathy Medication Market Competitive Analysis:

Some of the major companies in the global cardiomyopathy medication market include Pfizer Inc.,   Array Biopharma, Inc., PhaseBio Pharmaceuticals, Inc., Sanofi-Aventis US LLC, AstraZeneca, F. Hoffmann-La Roche Ltd, Capricor Therapeutics., Merck & Co., Inc., MyoKardia, Janssen Products, LP, Ionis Pharmaceuticals, Inc., Becton And Dickson & Co., Medtronic, Biomerieux, and Teva Pharmaceutical Industries Ltd.

Cardiomyopathy Medication Market Segmentation:

The cardiomyopathy medication market is segmented on type, treatment, and end user.

Based on type, the market has been segmented into hypertrophic cardiomyopathy, dilated cardiomyopathy, restrictive cardiomyopathy, unclassified cardiomyopathy, and arrhythmogenic right ventricular dysplasia.

By treatment, the global cardiomyopathy medication market has been categorized as antiarrhythmics, anticoagulants, cardiac glycosides, antihypertensives, and diuretics.

The end user segments of the market are hospitals and clinics, homecare, and others. Of these, the hospitals and clinics segment are expected to dominate the market during the review period.

Cardiomyopathy Medication Market Regional Analysis:

Regionally, the global cardiomyopathy medication market is segmented into the Americas, Asia-Pacific, Europe, and Middle East & Africa. Of these, the market in the Americas is relied upon to overwhelm the global cardiomyopathy medication market because of the expanding per capita human services consumption and nearness of an enormous number of social insurance organizations in the district. The market in Asia-Pacific is anticipated to enroll the most elevated development rate during the estimate time frame inferable from the expanding attention to heart maladies and expanding government activities for human services changes.

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Cardiomyopathy Medication Industry News:

In 2019, the U.S. Nourishment and Drug Administration affirmed Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) containers for the treatment of the coronary illness (cardiomyopathy) brought about by transthyretin interceded amyloidosis (ATTR-CM) in grown-ups.

In 2019, Researchers at the University of Arizona College of Medicine - Phoenix have appeared without precedent for preclinical investigations that Aliskiren, a medication that restrains the compound that manages pulse, can defer the movement of congestive cardiovascular breakdown and stretch endurance rates.

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CNS SPECIFIC ANTISENSE OLIGONUCLEOTIDE MARKET ANALYSIS

Antisense oligonucleotides are synthetic strings of nucleic acid, which reduce expression of messenger RNA (m-RNA). The function performed by interference with pre m- RNA splicing are restoration of protein and modification of protein. Antisense oligonucleotide is a new approach to treat several neurodegenerative disorders to prevent disease onset or stop disease development.

Market Dynamics

Increasing prevalence of neurodegenerative disease is a major factor driving CNS specific antisense oligonucleotide market growth. According to fact sheet from Alzheimer Association, an estimated around 5.7 million Americans were diagnosed with Alzheimer's dementia in 2018. Moreover, rich product pipeline for the treatment of CNS disorders and rare diseases associated with it is expected to boost the market growth. For instance, in December 2018, Biogen and Ionis Pharmaceuticals product IONIS-SOD1Rx (BIIB067) cleared Phase 1 clinical trial and is expected to be launched by 2020.

Key features of the study:

This report provides in-depth analysis of the CNS specific antisense oligonucleotide market and provides market size (US$ million) and Compound Annual Growth Rate (CAGR %) for the forecast period (2018 – 2026), considering 2017, as the base year

It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market

This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends,  regional outlook, and competitive strategy adopted by key players

It profiles key players in the global CNS specific antisense oligonucleotide market based on the following parameters – company overview, financial performance, product portfolio, market presence, distribution strategies, key developments, strategies, and future plans

Key companies covered as a part of this study include, Alnylam Pharmaceuticals Inc., Sarepta Therapeutics Inc., Biogen Inc., Ionis Pharmaceuticals Inc., Wave Life Sciences Ltd., Stroke Therapeutic Inc., Dynacure, ProQR Therapeutics N.V., Q-STATE BIOSCIENCES, INC.

Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, technology up-gradation, market expansion, and marketing tactics

The global CNS specific antisense oligonucleotide market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts

Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the CNS specific antisense oligonucleotide market

Detailed Segmentation:

Patisiran

Nusinersen

Inotersen

IONIS-HTT Rx (RG6042)

Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy

Spinal Muscular Atrophy

Huntington’s Disease

Hospital Pharmacy

Retail Pharmacy

Online Pharmacy

Patisiran

Nusinersen

Inotersen

IONIS-HTT Rx (RG6042)

Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy

Spinal Muscular Atrophy

Huntington’s Disease

Hospital Pharmacy

Retail Pharmacy

Online Pharmacy

U.S.

Canada

Patisiran

Nusinersen

Inotersen

IONIS-HTT Rx (RG6042)

Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy

Spinal Muscular Atrophy

Huntington’s Disease

Hospital Pharmacy

Retail Pharmacy

Online Pharmacy

Brazil

Mexico

Argentina

Rest of Latin America

Patisiran

Nusinersen

Inotersen

IONIS-HTT Rx (RG6042)

Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy

Spinal Muscular Atrophy

Huntington’s Disease

Hospital Pharmacy

Retail Pharmacy

Online Pharmacy

Germany

U.K.

France

Italy

Spain

Russia

Rest of Europe

Patisiran

Nusinersen

Inotersen

IONIS-HTT Rx (RG6042)

Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy

Spinal Muscular Atrophy

Huntington’s Disease

Hospital Pharmacy

Retail Pharmacy

Online Pharmacy

China

India

Japan

Australia

South Korea

ASEAN

Rest of Asia Pacific

Patisiran

Nusinersen

Inotersen

IONIS-HTT Rx (RG6042)

Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy

Spinal Muscular Atrophy

Huntington’s Disease

Hospital Pharmacy

Retail Pharmacy

Online Pharmacy

GCC

Israel

Rest of Middle East

Patisiran

Nusinersen

Inotersen

IONIS-HTT Rx (RG6042)

Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy

Spinal Muscular Atrophy

Huntington’s Disease

Hospital Pharmacy

Retail Pharmacy

Online Pharmacy

South Africa

Central Africa

North Africa

Company Overview

Product Portfolio

Financial Performance

Key Highlights

Market Strategies

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CNS Specific Antisense Oligonucleotide Market Development and Trends Forecasts Report 2018-2026

Antisense oligonucleotides are synthetic strings of nucleic acid, which reduce expression of messenger RNA (m-RNA). The function performed by interference with pre m- RNA splicing are restoration of protein and modification of protein. Antisense oligonucleotide is a new approach to treat several neurodegenerative disorders to prevent disease onset or stop disease development.

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Market Dynamics

Increasing prevalence of neurodegenerative disease is a major factor driving CNS specific antisense oligonucleotide market growth. According to fact sheet from Alzheimer Association, an estimated around 5.7 million Americans were diagnosed with Alzheimer's dementia in 2018. Moreover, rich product pipeline for the treatment of CNS disorders and rare diseases associated with it is expected to boost the market growth. For instance, in December 2018, Biogen and Ionis Pharmaceuticals product IONIS-SOD1Rx (BIIB067) cleared Phase 1 clinical trial and is expected to be launched by 2020.

Increasing prevalence of cancer is expected to propel growth of the CNS specific antisense oligonucleotide market

CNS specific antisense oligonucleotide is used for treatment of neurodegenerative disorders and associated rare diseases. According to February 2017, report of National Center for Biotechnology Information (NCBI), an estimated the prevalence of Cerebral palsy ranges from 1.5 to more than 4 per 1,000 of children from age 10 to 14 years. The birth prevalence of Cerebral palsy is around 2 per 1,000 live births globally.

Furthermore, launch of new products for rare disease associated with CNS disorders is propelling growth of the CNS specific antisense oligonucleotide market over the forecast period. For instance, in October 2018, Ionis Pharmaceuticals, Inc. and Akcea Therapeutics, Inc. received the U.S. Food and Drug Administration (FDA) approval for its novel drug TEGSEDITM (inotersen) indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. In the same year, company received approval from Canadian Health authority for the novel drug TEGSEDITM (inotersen).

However, high cost of treatment and risk of toxicity is expected to hinder the market growth. According to the Journal Molecular Therapy, in May 2017, Biogen priced Spinraza (nusinersen injection) at US$ 750,000 for the first year’s treatment (US$125,000 per injection) and US$350,000 per year.

Key Players

Major players operating in the global CNS specific antisense oligonucleotide market include Alnylam Pharmaceuticals Inc., Sarepta Therapeutics Inc., Biogen Inc., Ionis Pharmaceuticals Inc., Wave Life Sciences Ltd., Stroke Therapeutic Inc., Dynacure, ProQR Therapeutics N.V., and Q-STATE BIOSCIENCES, INC.

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Detailed Segmentation:

   Global CNS Specific Antisense Oligonucleotide Market, By Drug:

       Approved

          Patisiran

          Nusinersen

          Inotersen

       Pipeline

          IONIS-HTT Rx (RG6042)

   Global CNS Specific Antisense Oligonucleotide Market, By Indication:

       Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy

       Spinal Muscular Atrophy

       Huntington’s Disease

   Global CNS Specific Antisense Oligonucleotide Market, By Distribution Channel:

       Hospital Pharmacy

       Retail Pharmacy

       Online Pharmacy

   Global CNS Specific Antisense Oligonucleotide Market, By Geography:

       North America

Europe

Asia Pacific

Latin America

Middle East

Africa

Key questions answered in the report:

1. What will the market growth rate of market in 2026 2. What are the key factors driving the global market 3. Who are the key manufacturers in market space? 4. What are the market opportunities, market risk and market overview of the market? 5. What are sales, revenue, and price analysis by types and applications of market? 6. What are sales, revenue, and price analysis by regions of industry?

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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?

[rajputdishank20-blog]

Antisense and RNAi Therapeutics Market Sales, Trend, Region Forecast and Manufacturers in 2019-2025

Antisense technology provides the opportunity to manipulate the gene expression and this is being considered as an effective treatment for various diseases. This opportunity is being cashed on by the companies and are investing in R&D in this sector.

The possibility of studying of gene function for disease management by regulating the gene expression by the malfunctioning gene, R&D carried out with respect to treatment of uncommon neurodegenerative diseases, such as hereditary ATTR amyloidosis, are some of the other factors anticipated to drive the antisense and rnai therapeutics market.

Read More: https://www.marketwatch.com/press-release/antisense-and-rnai-therapeutics-market-sales-trend-region-forecast-and-manufacturers-in-2019-2025-2019-12-18

[market-insights-dbmr]

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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[healthcaretrends-blog]

CNS Specific Antisense Oligonucleotide Market Study 2019: Impressively growing Opportunities

Antisense oligonucleotide plays a major role in modern healthcare, as it acts on the messenger RNA level before the protein is formed to inhibit expression of certain diseases-causing genes.

The global CNS specific antisense oligonucleotide market size was valued at US$ 883.7 million in 2017, and is expected to exhibit a CAGR of 24.9% over the forecast period (2018 – 2026).

CNS specific antisense oligonucleotide is used for treatment of neurodegenerative disorders and associated rare diseases. According to February 2017, report of National Center for Biotechnology Information (NCBI), an estimated the prevalence of Cerebral palsy ranges from 1.5 to more than 4 per 1,000 of children from age 10 to 14 years. The birth prevalence of Cerebral palsy is around 2 per 1,000 live births globally.

Furthermore, launch of new products for rare disease associated with CNS disorders is propelling growth of the CNS specific antisense oligonucleotide market over the forecast period. For instance, in October 2018, Ionis Pharmaceuticals, Inc. and Akcea Therapeutics, Inc. received the U.S.  Food and Drug Administration (FDA) approval for its novel drug TEGSEDITM (inotersen) indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. In the same year, company received approval from Canadian Health authority for the novel drug TEGSEDITM (inotersen).

However, high cost of treatment and risk of toxicity is expected to hinder the market growth. According to the Journal Molecular Therapy, in May 2017, Biogen priced Spinraza (nusinersen injection) at US$ 750,000 for the first year’s treatment (US$125,000 per injection) and US$350,000 per year.

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North America holds the dominant position in the global CNS specific antisense oligonucleotide market, owing to strong government support for antisense drugs. For instance, in March 2018, GeneTx Biotherapeutics LLC’s, GTX-101 received the U.S. Food and Drug Administration’s (FDA) orphan-drug designation for GTX-101 for the treatment of Angelman syndrome. It is a rare neurogenetic disorder that affects around one in 15,000 people, according to the FDA statement.

Increasing prevalence of Alzheimer disease in the region is also expected to boost the market growth. For instance, in August 2012, National Center for Biotechnology Information (NCBI) stated Alzheimer disease as the most frequent cause of dementia in Western societies and estimated around 5.5 million cases in the U.S. and the prevalence worldwide is estimated to be as high as 24 million. Moreover, rich product pipeline for treatment of neurodegenerative disorders is expected to drive the market growth. For instance, in October 2017, Ionis Pharmaceuticals, Inc., initiated a Phase 1/2 clinical study of IONIS-MAPTRx in patients with mild Alzheimer's disease. Biogen funded US$ 10 million milestone payment to Ionis Pharmaceuticals, Inc.

Moreover, key players in the players are focused on receiving the U.S. Food and Drug Administration (FDA) approval for novel products to reduce the burden of CNS associated disorders. For instance, in September 2016, Sarepta Therapeutics received the U.S. Food and Drug Administration (FDA) approval for Exondys 51(eteplirsen) used for treatment of rare genetic disorder Duchenne muscular dystrophy.

Key Players

Major players operating in the global CNS specific antisense oligonucleotide market include Alnylam Pharmaceuticals Inc., Sarepta Therapeutics Inc., Biogen Inc., Ionis Pharmaceuticals Inc., Wave Life Sciences Ltd., Stroke Therapeutic Inc., Dynacure, ProQR Therapeutics N.V., and Q-STATE BIOSCIENCES, INC.

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[coolluminaryland-blog1]

Amyloidosis Treatment Market : Global Segments, Top Key Players, Size and Recent Trends by Forecast to 2025

Amyloid is an abnormal protein produced in the bone marrow. Amyloidosis is a rare disease caused by extracellular and intracellular deposition of amyloid which alters the normal activities of the tissue. Commonly affected organs by amyloidosis encompass kidney, heart, nervous system, digestive tract, liver and spleen. Severe form of amyloidosis in later stages can lead to permanent organ failure and in extreme cases can be life-threatening also. There are over 30 different types of amyloid proteins discovered so far and treatment can only help in managing the symptoms and regulating the production of amyloid protein and not the permanent cure.

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About 90% of the amyloid formed due to the aggregation of misfolded proteins whereas, 10% of amyloidosis is due to presence of components such as apolipoprotein-E (apoE), glycosaminoglycans (GAGs) and serum amyloid P-component (SAP). According to American Society of Hematology the prevalence rate of amyloidosis in the United States is approximately 1 in 90,666 of which 60 – 65 % of the patient are men. Prominent symptoms of the disease include fatigue, weight loss, breathlessness, peripheral oedema, carpal tunnel syndrome, postural hypotension and sensory change. In the United Kingdom systemic amyloidosis is responsible for approximately one in 1,200 deaths each year.

Amyloidosis can be broadly classified into 3 major categories namely AA amyloidosis, AL amyloidosis and Transthyretin amyloidosis. In general amyloidoses are generally denoted by a capital “A” signifying the amyloid present followed by an abbreviation for the fibril protein. AA amyloidosis is a normal sequence serum amyloid – A protein produced mainly in the liver in response to multiple cytokines. AA mostly occur in various chronic inflammatory disorders and is one of the most common systemic amyloidosis across the globe. Some of the conditions associated with AA include: rheumatoid arthritis, alzheimer disease, psoriasis, tuberculosis, renal cell carcinoma, hodgkin disease and non-hodgkin lymphoma.

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AL amyloidosis or light chain amyloidosis monoclonal plasma cell disorder closely related to multiple myeloma. Organs involved in the AL amyloidosis include heart, kidney, peripheral nerve, gastrointestinal tract, respiratory tract and other organs. Treatment for AL amyloidosis in most instances is similar to multiple myeloma i.e. chemotherapy. Selected patient are treated with high-dose melphalan and autologous stem-cell transplantation. Alternative therapy includes rituximab, etanercept, thalidomide, iododoxorubicin and lenalidomide. In about about 80% the cases AA amyloidosis is fetal with median survival rate of 1-2 years only. Transthyretin amyloidosis (ATTR) is one of the most frequent inherited systemic forms of amyloidosis. ATTR is an autosomal-dominant disease but is associated with at least 100 different transthyretin mutations. Other forms of amyloidosis include heavy-chain amyloidosis, beta2 -microglobulin amyloidosis, hereditary renal amyloidosis and Central nervous system amyloidosis.

Major drivers for the growth of the global amyloidosis market include favourable investment and reimbursement facilities in developed economic countries. On the other hand high cost involved in treatment and limited awareness may hinder the growth of the overall market. Biopsy is the widely accepted diagnostic tool for amyloidosis and in about two-third of the patient alkylator-based chemotherapy is effective in managing amyloidosis. 

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Noninvasive tests such as echocardiography of the heart and X-ray of bone abnormalities may provide supportive information but are not sufficient for the diagnosis of amyloidosis. Some of the prominent players operating in global amyloidosis market include Pfizer Inc., GlaxoSmithKline plc, Celgene Corporation, Alnylam Pharmaceuticals, Inc., Takeda Pharmaceutical Company Limited, Prothena Corporation plc. Premier institutes and universities promoting the development of amyloidosis clinical trial studies include Stanford University, Boston University, IRCCS Policlinico San Matte and Umea University.

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