Hereditary Transthyretin Amyloidosis Competitive Landscape And Market

DelveInsight’s ‘Hereditary Transthyretin Amyloidosis (hATTR) - Market Insights, Epidemiology and Market Forecast– 2030’ report delivers an in-depth understanding of the hATTR, historical and forecasted epidemiology as well as the hATTR market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

 

The hATTR market report provides current treatment practices, emerging drugs, and market share of the individual drugs, current and forecasted 7MM hATTR market size from 2018 to 2030. The report also covers current hATTR treatment algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered

  • The United States
  • EU5 (Germany, Spain, Italy, France, and United Kingdom)
  • Japan

Study Period: 2018–2030

hATTR Disease Understanding and Treatment Algorithm

hATTR Overview

Transthyretin (earlier known as prealbumin) is an abundant, soluble, β-strand rich 55 kDa homotetramer serum protein that is responsible for the transportation of both vitamin A (via retinol-binding protein) and thyroxin throughout the body. TTR is also involved in the binding and redistribution of β-amyloid in the choroid plexus as well as in the retention of T4 in the cerebral spinal fluid (CSF). TTR may sometimes dissociate into its 127-amino acid monomeric subunits and undergo aberrant changes to form amyloidogenic intermediates. These intermediates then might self-associate to become amyloid fibrils that accumulate as amyloid deposits throughout the body, resulting in Transthyretin Amyloidosis.  Transthyretin Amyloidosis (ATTR) can be sub-classified as wild-type (wt) or hereditary, and the latter is then further sub-divided into familial amyloid polyneuropathy (FAP) and familial amyloid cardiomyopathy (FAC).

 

The hereditary forms of amyloidosis are autosomal dominant diseases characterized by deposition of variant proteins, in distinctive tissues. The most common hereditary form is transthyretin amyloidosis (ATTR) caused by the misfolding of protein monomers derived from the tetrameric protein transthyretin (TTR). Mutations in the gene for TTR frequently result in instability of TTR and subsequent fibril formation. Closely related is wild-type TTR in which the native TTR protein, particularly in the elderly, can destabilize and re-aggregate causing nonfamilial cases of TTR amyloidosis.

 

Familial transthyretin amyloidosis (FTA) is caused by changes (mutations) in the TTR gene. This gene is responsible for making a protein called transthyretin which transports vitamin A and a hormone called thyroxin too many parts of the body. Mutations in TTR lead to a transthyretin protein that is not made correctly. The faulty protein then folds up to form amyloid. Amyloid builds up in various parts of the body causing nerve and tissue damage. Most people who have FTA have inherited the TTR mutation from a family member. However, a few people with FTA will have no family history of the disease and have a new (de novo) mutation in the TTR gene.

 

hATTR Diagnosis

Disease heterogeneity and its rarity make a diagnosis of hATTR amyloidosis challenging. However, making a correct diagnosis is vital to determining prognosis, treatment, and appropriate patient and family counseling. Timely diagnosis is also important because it allows patients the opportunity to receive appropriate care as early as possible in the disease course.

 

Diagnosis can be confirmed via biopsy of the affected tissue or organ followed by staining with Congo red to confirm the presence of amyloid. Diagnosis can be established less invasively through biopsy of the salivary gland, endoscopic biopsy of the gastric mucosa, or subcutaneous fat aspiration. Western blot analysis, immunohistochemical staining, laser microdissection, proteomics, and mass spectrometry are subsequently used to characterize amyloid type. Limitations of biopsy are due to the often patchy distribution of amyloid deposits, sometimes necessitating multiple biopsies to confirm or exclude the diagnosis. Additionally, biopsy sensitivity depends on multiple factors, such as pathologist experience and protocol for Congo red staining. In patients with a family history of disease and/or evaluation of symptomatic burden (ie, polyneuropathy), genetic testing is a crucial component to confirm a hATTR amyloidosis diagnosis as it identifies the specific TTR mutation present. Presymptomatic testing is now widely available and may be performed at the request of the patient with appropriate genetic counseling and follow-up.

 

Initial laboratory testing should include a complete blood count with red cell indices, HPLC or Hb electrophoresis, and eventually α/β-globin chain synthesis ratio measurement. The latter procedure, however, is sometimes bypassed by DNA analysis as a less complicated method to diagnose α- thalassemia.

 

hATTR Treatment

Current treatment options for patients with TTR amyloidosis are limited, with only symptomatic treatment and transplantation. For patients diagnosed with TTR-FAP who have the mild or moderate disease and confirmed by genetic testing and biopsy, a liver transplant is the current standard of care. However, symptomatic treatment is used to provide immediate relief.

 

Since variant TTR is mainly produced in the liver, transplanting a new liver should almost eliminate the production of variant protein and further halt the disease progression outside the brain and eyes. However, liver transplant does not effectively prevent cardiomyopathy in most cases and is not recommended for patients with late-stage TTR-FAP or with leptomeningeal-type amyloidosis. Thus, for these patients, symptomatic relief is the only available treatment. The very first pharmacologic agent to receive approval for the treatment of TTR-FAP was in 2011 and many other agents are in various stages of development.

hATTR Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of hATTR, diagnosed prevalent cases of hATTR, type-specific cases of hATTR, stage-specific cases of hATTR, and New York Heart Association (NYHA) classification of familial amyloid cardiomyopathy (FAC) in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan market from 2018 to 2030.

 

Key Findings

  • This section provides glimpse of the hATTR epidemiology in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.
  • The total diagnosed prevalent cases of hATTR in the 7MM were estimated to be 13,540 in 2020 growing at a CAGR of 5.05% during the study period (2018–2030).
  • Epidemiology assessed for the condition showed that the US, in 2020, accounted for approximately 15,312 prevalent cases of hATTR.
  • Among the EU-5 countries in 2020, France had the highest diagnosed prevalent cases of hATTR with 1,480 cases. In contrast, Germany had the lowest cases (518) in 2020.
  • Japan accounted for 648 diagnosed prevalent hATTR cases in 2020.
  • In the US, in 2020, the highest proportion of type-specific cases were observed in Familial Amyloid Polyneuropathy (FAP) in comparison to Familial Amyloid Cardiomiopathy (FAC) and Mixed hATTR Type.
  • In the EU-5, NYHA Class II (748) accounted for the highest cases in 2020, followed by NYHA Class III (303) and NYHA Class I (175).
  • Among the stage-specific diagnosed prevalent contribution, in Japan stage 3 cases were observed to be 47 and stage 1 cases were 180 in 2020.
  • The United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan – hATTR Epidemiology

hATTR Drug Chapters

The drug chapter segment of the hATTR report encloses the detailed analysis of hATTR current treatment pattern and pipeline drugs. It also helps to understand the hATTR clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drugs and the latest news and press releases.

 

hATTR Marketed Drugs

 

Vyndaqel: Pfizer

Vyndaqel (tafamidis meglumine 20 mg) and Vyndamax (tafamidis 61 mg) are oral transthyretin stabilizers indicated for the treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. Tafamidis is a selective stabilizer of transthyretin. Tafamidis binds to transthyretin at the thyroxine-binding sites, stabilizing the tetramer and slowing dissociation into monomers, the rate-limiting step in the amyloidogenic process.

 

Previously, in November 2012, the US FDA had accepted for review the New Drug Application (NDA) for tafamidis meglumine for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) along with granting the tafamidis NDA a priority-review designation and had also provided an anticipated Prescription Drug User Fee Act (PDUFA) action date as June 2012.

Product details in the report…

 

Onpattro: Alnylam Pharmaceuticals

Onpattro (patisiran) contains a transthyretin-directed small interfering RNA and is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Patisiran is a double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference, which reduces serum TTR protein and TTR protein deposits in tissues. It is supplied as a sterile, preservative-free, and white to off-white, opalescent, homogeneous solution for intravenous infusion in a single-dose glass vial.

 

The drug is currently undergoing Phase III APOLLO-B study evaluating the drug in participants with ATTR with cardiomyopathy. The APOLLO-B study completed enrollment in June 2021 and is expected to have topline data available in mid-2022. Based on those data and subsequent regulatory interactions, the potential path forward for a HELIOS-B Phase III study of another investigational vutrisiran in patients with transthyretin-mediated amyloidosis with cardiomyopathy-interim analysis will be further refined, including the potential for an earlier readout of topline results.

Product details in the report…

 

Tegsedi: Akcea Therapeutics/Ionis Pharmaceuticals

Tegsedi (inotersen) is a transthyretin-directed antisense oligonucleotide. Tegsedi is specifically indicated for the treatment of the hATTR-PN in adults. Tegsedi is supplied as a solution for subcutaneous injection. Due to safety concerns of Tegsedi (thrombocytopenia and glomerulonephritis), the drug is available only through REMS (Risk Evaluation and Mitigation Strategy) programme.

 

Recommended dose: 284 mg injected subcutaneously once weekly. For dosing consistency, patients should be instructed to give the injection on the same day every week. If a dose is missed, patients should be instructed to take the missed dose as soon as possible, unless the next scheduled dose is within 2 days. In this situation, the patient should be directed to skip the missed dose and take the next scheduled dose on the scheduled day.

Product details in the report…

 

hATTR Emerging Drugs

 

Vutrisiran: Alnylam Pharmaceuticals

Vutrisiran is an investigational, subcutaneously-administered RNAi therapeutic in development to treat ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and mutant transthyretin (TTR) protein before it is made.

 

Quarterly and potentially biannual administration of vutrisiran may help reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s next-generation delivery platform known as the Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections. The drug is currently in the Phase III stage of development.

Product details in the report…

 

Eplontersen: Ionis Pharmaceuticals/ AstraZeneca

Eplontersen, formerly known as ION-682884, IONIS-TTR-LRx, and AKCEA-TTR-LRx, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce the production of transthyretin, or TTR protein, to treat all types of ATTR. In patients with ATTR, both the mutant and wild-type TTR protein builds up as fibrils in tissues, such as the peripheral nerves, heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid, and bone marrow. The presence of TTR fibrils interferes with the normal functions of these tissues. As the TTR protein fibrils enlarge, more tissue damage occurs, and the disease worsens, resulting in poor quality of life and eventually death.

 

The drug is currently advancing into the Phase III stage of clinical development activity for hATTR polyneuropathy and ATTR cardiomyopathy, with data readout expected in 2022 and 2024, respectively. The company has achieved complete enrollment in the Phase III NEURO-TTRansform study of eplontersen in patients with TTR polyneuropathy and is expected to release data by mid-2022.

Product details in the report…

 

Acoramidis (AG 10): Eidos Therapeutics

Licensed from Stanford University in 2016, acoramidis (formerly AG10) is an investigational, orally-administered small molecule being developed by Eidos Therapeutics (a subsidiary of BridgeBio Pharma), designed to potently stabilize tetrameric transthyretin or TTR, thereby halting at its outset the series of molecular events that give rise to TTR amyloidosis, or ATTR. AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a rescue mutation because co-inheritance has been shown to prevent or ameliorate ATTR in individuals also inheriting a pathogenic or disease-causing mutation in the TTR gene. AG10 is the only TTR stabilizer in development that has been observed to mimic the stabilizing structure of this rescue mutation.

 

Acoramidis is currently being studied in a Phase III clinical trial in patients with ATTR-CM (ATTRibute-CM) and a Phase III clinical trial in patients with ATTR-PN (ATTRibute-PN). The company has reported the enrollment of 632 participants in the ATTRibute-CM Phase III study and anticipates top-line data to be released in 4Q 2021 or early 2022; for Part B, the top-line data is expected to be published in 2023. It expects the filing for NDA submission and MAA for Phase III clinical trial in patients with ATTR-CM (ATTRibute-CM) in mid- 2022 and the launch of top-line data for acoramidis (Phase III clinical trials) in patients with ATTR-PN (ATTRibute-PN) in 2024.

Product details in the report…

 

CRX-1008 (Tolcapone; SOM0226): Corino Therapeutics

CRX-1008 (also known as Tolcapone; SOM0226) is an investigational and potent small molecule stabilizer of TTR that is under development by Corino Therapeutics in the Phase II stage of development for the treatment of familial amyloid polyneuropathy (FAP). It is a repositioned compound that acts by imitating the process of binding thyroxine to TTR in the bloodstream and is administered via the oral route. It inhibits TTR tetramer dissociation into monomeric TTR and prevents the accumulation of amyloid in various tissues. CRX-1008 has unique and differentiated activity compared to other kinetic stabilizers, including the ability to cross an intact blood-brain barrier.

 

The company has completed multiple clinical proofs of concept studies to evaluate the safety, tolerability, and TTR stabilization activity of CRX-1008. Data from these studies are expected in 2021, and the company further plans for Phase II/III studies.

Product details in the report…

hATTR Market Outlook

Hereditary Transthyretin Amyloidosis (hATTR) market has been assessed, based on demand, prescription analysis and the annual cost of therapy of current and forecasted market value of the approved drugs, Vyndaqel, Onpattro, Tegsedi in the US, Europe, and Japan as well as forecasted patient share and the annual cost of therapy for upcoming medicines. Supportive therapies like Diflunisal, other symptomatic drugs for TTR-FAP and recommended symptomatic drugs like loop diuretics, aldosterone antagonists, angiotensin-converting enzyme inhibitors, and beta-blockers have been considered for hATTR market estimation. Liver transplantation is the most common treatment method in hATTR amyloidosis, as the liver is the main source of abnormal TTR production. This procedure is most effective in patients who are in the early stages of the disease.

 

In addition, only a few therpaies have been approved for this indication. Each drug has a different purpose or mode of action, such as stabilizing the TTR protein, preventing the production of the TTR protein, or removing amyloid deposits. The market scenario started changing in 2018, with the launch of Tegsedi and Onpattro, and witnessed further growth in 2019, with the US launch of Vyndaqel/Vyndamax.

 

Tegsedi (inotersen) is a transthyretin-directed antisense oligonucleotide. In October 2018, Akcea Therapeutics and Ionis Pharmaceuticals announced the US FDA approval of the drug for the treatment of hATTR-PN. Previously, in July 2018, Tegsedi had also received approval from the EC for stage I or stage II in adult patients with hATTR-PN. Use of Inotersen has demonstrated significant benefit in Norfolk Quality of Life Questionnaire-Diabetic Neuropathy and modified Neuropathy Impairment Score +7. However, the drug has not been approved by the PMDA, Japan.

 

Both Tegsedi and Onpattro have received Fast Track Designation and Orphan Drug Designation for Transthyretin Amyloidosis. Onpattro has additionally received Breakthrough Therapy Designation from the FDA for the treatment of hATTR-PN. Onpattro is projected to hold a significant share of the hATTR-PN market with good safety and efficacy results even for the late stage of the hATTR-PN and may grab a large portion of the market if the cardiac population is penetrated for treatment. Tegsedi's performance is not good, and onpattro is outperforming this candidate. Hence, it will not generate significant revenue. Ionis’ emerging candidate Eplontersen, however seems to be good contender in the future for hATTR.

 

Even though the current hATTR treatment options are limited, multiple potential therapies are emerging to help mitigate the underlying genetic mutation in patients with hATTR amyloidosis. Major potential emerging drugs are Vutrisiran, and Eplontersen

 

Alnylam’s Vutrisiran is an investigational, subcutaneously-administered RNAi therapeutic. Alnylam has completed enrollment in its HELIOS-B Phase III study in patients with hATTR-CM. The company is expected to report 30-month endpoint top-line results from the HELIOS-B trial study in early 2024. The US FDA is currently evaluating the NDA for the drug to treat hATTR-PN. The approval of the drug for both segments of the patient will help the company grab a significant share in the market of hATTR.

 

Eidos Therapeutics’ orally available, small molecule TTR stabilizer, AG 10, is another molecule that has demonstrated potent activity in Phase II clinical trial. The company anticipates data to be released in 4Q 2021 or early 2022. The filing for NDA submission and MAA is anticipated in mid- 2022 (Bridgebio, 2021). The company is expecting top-line data for acoramidis for Phase III clinical trials in patients with ATTR-PN (ATTRibute-PN) in 2024. In addition, BridgeBio expects to submit an application for regulatory approval of the drug in 2022 to the FDA. In addition, Alexion holds an exclusive license to develop and commercialize AG10 in Japan. It is conducting a Phase III bridging study of ALXN2060 exclusively in Japan for patients with ATTR-CM. The study is expected to be completed in April 2023.

 

Key Findings

  • The market size of hATTR in the seven major markets is expected to rise from USD 973.5 million in 2020 during the study period (2018–2030).
  • Current market dynamics comprises approved therapies like Tafamidis, Inotersen, Patisiran (Onpattro), and other off-label therapies.
  • Potential Therapies expected to launch are as follows Vutrisiran (Alnylam Pharmaceuticals), Eplontersen (AKCEA-TTR-LRx) (Ionis Pharmaceuticals), Acoramidis (Eidos Therapeutics), CRX-1008 (Tolcapone) (Corino Therapeutics), PRX004 (Prothena/Novo Nordisk), and others. The launch of these therapies may increase market size in the coming years, assisted by an increase in the hATTR patient pool.
  • The United States accounts for the largest market size of hATTR compared to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan. In the United States, the market size of hATTR is anticipated to rise from USD 529.6 million in 2020 during the forecast period.
  • Among the EU5 countries, France had the largest market size (USD 125.0 million) in 2020, while Germany had the smallest with USD 44.8 million.
  • In 2020, Japan accounted for a market size of USD 77.1 million.

Analyst views

  • Patients typically face difficulty getting treatment, since there are only a few Amyloid Institutes of Excellence at academic medical centers. In addition, existing off-label treatments are ineffective. As a result, patients and their families prefer therapies with patient-friendly RoA.  Drugs with improved safety and efficacy, along with a convenient mode of administration, may become popular and successful in the future.
  • Onpattro's sales revenue is higher than Tegsedi's. Real-world evidence substantially favors Onpattro over Tegsedi. Tegsedi was licensed by the FDA in 2018 to treat hATTR polyneuropathy, however, sales have lagged below expectations. Even though Tegsedi is a subcutaneous alternative for hATTR with polyneuropathy, it is a weekly injectable with black box warnings for adverse effects, along with mandating regular monitoring has led to its lower than expected sales.
  • Vyndaqel/Vyndamax had a strong launch in the United States, and it is expected to grab a lion market share for the hATTR cardiomyopathy segment. While Vyndaqel was just recently licensed in the United States, it has been approved in Europe since 2011 under the trade name Vyndamax for the treatment of hATTR neuropathy. We do not expect Vyndaqel/Vyndamax will be approved for PN in the United States, based on the evidences. Furthermore, we believe the company has moved its attention to CM patients because the market for PN is almost saturated (in the EU5 and Japan) for a drug that has been available for almost a decade in these regions, thereby expected to get impacted by the launch of emerging therapies for PN.
  • In the future, patients will have a choice of approved therapies, thanks to the entry of pipeline therapies in the hATTR market.  There are a variety of drugs in the pipeline, ranging from monoclonal antibodies to ASOs to small molecules, and CRISPR therapy. Products with various mechanisms might be highly beneficial to patients while also presenting a profitable opportunity for drug developers. 
  • Among the emerging therapies, Vutrisiran, a subcutaneous formulation from Alnylam, and Eplontersen, a subcutaneous formulation from Ionis, are expected to hit the market shortly. Eplontersen, Ionis' next-generation drug, is already in clinical trials and might be a competitor to Alnylam's Vutisiran, and Onpattro. However, it will require a monthly dosage. With TEGSEDI's sales revenue falling short of projections, Ionis has high hopes for Eplontersen. Vutrisiran, on the other hand, is administered subcutaneously every three months, which is more convenient for patients. The company also intends to have a bi-annual dosing regimen.
  • This section provides the total hATTR market size and market size by drug in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

hATTR Drug Uptake

This section focusses on the rate of uptake of the potential drug launched in the hATTR market or expected to get launched in the market during the study period 2018–2030. The analysis covers hATTR market uptake by drug; patient uptake by drug; and sales of each drug.

 

This helps in understanding the drug with the most rapid uptake, reasons behind the maximal use of new drug and allow the comparison of the drug on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

hATTR Development Activities

The report provides insights into different candidates in phase II, and phase III stage. It also analyzes key players involved in developing targeted drug.

 

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for hATTR emerging drug.

Competitive Intelligence Analysis

We perform competitive and market intelligence analysis of the hATTR market by using various competitive intelligence tools that include–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 hATTR, explaining its causes, signs and symptoms, pathogenesis and currently available drug.
  • Comprehensive insight has been provided into the hATTR epidemiology and treatment.
  • Additionally, an all-inclusive account of both the current and emerging drug for hATTR are provided, along with the assessment of new drug, which will have an impact on the current hATTR treatment landscape.
  • A detailed review of hATTR market; historical and forecasted is included in the report, covering the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan drug outreach.
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan hATTR market.

Report Highlights

In the coming years, hATTR 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 hATTR R&D. The drug under development are focused on novel approaches to treat/improve the disease condition.

 

Delvelnsight has analysed the total age-specific and total vaccinated population of hATTR.

hATTR Report Insights

  • Patient Population
  • Therapeutic Approaches
  • hATTR Pipeline Analysis
  • hATTR Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Drug

hATTR Report Key Strengths

  • Ten Years Forecast
  • The United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan Coverage
  • hATTR Epidemiology Segmentation
  • Key Cross Competition
  • Highly Analyzed Market
  • Drug Uptake

hATTR Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers

Key Questions

Market Insights:

  • What was the hATTR market share (%) distribution in 2018 and how it would look like in 2030?
  • What would be the hATTR total market size as well as market size by drug in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan during the forecast period (2021–2030)?
  • What are the key findings pertaining to the market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan?
  • At what CAGR, the hATTR market is expected to grow at the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan level during the forecast period (2021–2030)?
  • What would be the hATTR market outlook in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), aduring the forecast period (2021–2030)?
  • What would be the hATTR 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?

 

Epidemiology Insights:

  • What is the disease risk, burden and unmet needs of hATTR?
  • What is the historical hATTR patient pool in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan?
  • What would be the forecasted vaccinated pool of hATTR at the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), level?
  • What will be the growth opportunities in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan with respect to the patient population pertaining to hATTR?
  • At what CAGR the population is expected to grow in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan during the forecast period (2021–2030)?

 

Current Treatment Scenario, Marketed Drug and Emerging Drug:

  • What are the current options for the treatment of hATTR along with the approved drug?
  • What are the current treatment guidelines for the prevention of hATTR in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan?
  • What are the hATTR marketed drug and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
  • How many companies are developing drug for the treatment of hATTR?
  • How many drug are developed by each company for the treatment of hATTR?
  • How many emerging drug are in the mid-stage and late stage of development for the treatment of hATTR?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the hATTR drug?
  • What are the recent novel drug, targets, mechanisms of action and technologies developed to overcome the limitation of existing drug?
  • What are the clinical studies going on for hATTR and their status?
  • What are the key designations that have been granted for the emerging drug for hATTR?
  • What is the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan historical and forecasted market of hATTR?

Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the hATTR.
  • To understand the future market competition in the hATTR market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for hATTR in the United States, EU5 (Germany, Spain, Italy, France, and 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 hATTR market.
  • To understand the future market competition in the hATTR market.

1. Key Insights

2. Report Introduction

3. Hereditary Transthyretin Amyloidosis (hATTR) Market Overview at a Glance

3.1. Market Share by Therapies (%) Distribution of Hereditary Transthyretin Amyloidosis (hATTR) in 2020

3.2. Market Share by Therapies (%) Distribution of Hereditary Transthyretin Amyloidosis (hATTR) in 2030

4. Executive Summary of Hereditary Transthyretin Amyloidosis (hATTR)

5. Key Events

6. SWOT Analysis

7. Disease Background and Overview

7.1. Introduction

7.2. Familial amyloid cardiomyopathy (FAC)

7.3. Familial amyloid polyneuropathy (FAP)

7.4. Causes

7.5. Clinical Features

7.6. Symptom management of TTR-FAP

7.6.1. Staging overview (based on Coutinho)

7.6.1.1. Stage 0 disease

7.6.1.2. Stage I disease

7.6.1.3. Stage II disease

7.6.1.4. Stage III disease

7.6.2. Liver transplant recipients

7.6.3. DLT recipients

7.7. Pathophysiology

7.8. Genetics

7.9. Diagnosis and Monitoring

7.9.1. Tissue biopsy

7.9.1.1. Serum variant TTR protein

7.9.1.2. Genetic confirmation

7.9.2. Exclusionary diagnoses

7.9.2.1. AL amyloidosis

7.9.2.2. Mimicking neuropathies

7.9.2.3. Chronic inflammatory demyelinating polyneuropathy (CIDP)

7.9.2.4. Other exclusions

7.9.3. Tests for neuropathic symptoms

7.9.4. Tools for evaluating TTR-FAP progression

7.9.5. Scoring systems

7.9.6. Diseases with symptoms that overlap with that of hATTP amyloidosis:

7.9.7. Red flag symptoms associated with hATTR Amyloidosis:

7.9.8. Confirmation of hATTR Amyloidosis diagnosis:

7.9.9. Tools for evaluating TTR-FAP progression

8. Treatment

8.1. Disease-modifying treatments for TTR-FAP

8.2. Liver transplant

8.2.1. Outcomes of liver transplant for patients with TTR-FAP

8.2.1.1. Domino liver transplant (DLT)

8.2.1.2. Combined heart and liver transplant

8.3. Treatment Guidelines

8.3.1. First European Consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy

8.3.2. TTP stages according to symptom severity:

8.3.3. Algorithm for treatment of TTR-FAP

9. Epidemiology and Patient Population

9.1. Key Findings

9.2. Epidemiology Methodology

9.3. Total Prevalent Population of hATTR in the 7MM

9.4. Total Diagnosed Prevalent Population of hATTR in the 7MM

9.5. Assumptions and Rationale

9.6. The United States

9.6.1. Total Prevalent Population of hATTR in the United States

9.6.2. Total Diagnosed Prevalent Population of hATTR in the United States

9.6.3. Type-specific Cases of hATTR in the United States

9.6.4. Stage-specific Cases of Familial Amyloid Polyneuropathy (FAP) in the United States

9.6.5. New York Heart Association (NYHA) Classification of Familial Amyloid Cardiomyopathy (FAC) in the United States

9.7. EU5

9.7.1. Total Prevalent Population of hATTR in EU5

9.7.2. Total Diagnosed Prevalent Population of hATTR in EU5

9.7.3. Type-specific Cases of hATTR in EU5

9.7.4. Stage-specific Cases of Familial Amyloid Polyneuropathy (FAP) in EU5

9.7.5. New York Heart Association (NYHA) Classification of Familial Amyloid Cardiomyopathy (FAC) in EU5

9.8. Japan

9.8.1. Total Prevalent Population of hATTR in Japan

9.8.2. Total Diagnosed Prevalent Population of hATTR in Japan

9.8.3. Type-specific Cases of hATTR in Japan

9.8.4. Stage-specific Cases of Familial Amyloid Polyneuropathy (FAP) in Japan

9.8.5. New York Heart Association (NYHA) Classification of Familial Amyloid Cardiomyopathy (FAC) in Japan

10. Organizations contributing toward hereditary transthyretin amyloidosis (hATTR)

11. Patient Journey

12. Marketed Drugs

12.1. Key Competitors

12.2. Vyndaqel/Vyndamax: Pfizer

12.2.1. Product description

12.2.2. Regulatory milestones

12.2.3. Other development activities

12.2.4. Safety and Efficacy

12.2.5. Product Profile

12.3. Onpattro (patisiran): Alnylam Pharmaceuticals

12.3.1. Product description

12.3.2. Regulatory Milestones

12.3.3. Other development activities

12.3.4. Clinical Development activity

12.3.5. Safety and Efficacy

12.3.6. Product Profile

12.4. Tegsedi: Akcea Therapeutics/Ionis Pharmaceuticals

12.4.1. Product Description

12.4.2. Regulatory Milestones

12.4.3. Other Development Activities

12.4.4. Clinical Development activity

12.4.5. Safety and Efficacy

12.4.6. Product Profile

13. Emerging Drugs

13.1. Key Competitors

13.2. Vutrisiran: Alnylam Pharmaceuticals

13.2.1. Drug Description

13.2.2. Other Development Activities

13.2.3. Clinical development

13.2.4. Safety and Efficacy

13.3. Eplontersen: Ionis Pharmaceuticals/ AstraZeneca

13.3.1. Drug Description

13.3.2. Other Development Activity

13.3.3. Clinical development

13.3.4. Safety and Efficacy

13.4. Acoramidis (AG 10): Eidos Therapeutics

13.4.1. Drug Description

13.4.2. Other Development Activities

13.4.3. Clinical development

13.4.4. Safety and Efficacy

13.5. CRX-1008 (Tolcapone; SOM0226): Corino Therapeutics

13.5.1. Drug Description

13.5.2. Other Development Activities

13.5.3. Clinical Development

13.5.4. Safety and Efficacy

13.6. PRX004: Prothena/ Novo Nordisk

13.6.1. 1.5.1 Drug Description

13.6.2. Clinical Development

13.6.3. Safety and Efficacy

13.7. NTLA-2001: Intellia Therapeutics/Regeneron Pharmaceuticals

13.7.1. Drug description

13.7.2. Other development activities

13.7.3. Clinical development

13.7.4. Safety and Efficacy

14. Hereditary Transthyretin Amyloidosis (hATTR): Seven Major Market Analysis

14.1. Key Findings

14.2. Market Methodology

14.3. Total Market Size of hATTR in the 7MM

14.4. Market Size of hATTR by Current therapies in the 7MM

14.5. Market Size of hATTR by Emerging therapies in the 7MM

14.6. Market Outlook

14.7. Key Market Forecast Assumptions

14.8. United States Market Size

14.8.1. Total Market Size of hATTR in the United States

14.8.2. Market Size of hATTR by Current therapies in the United States

14.8.3. Market Size of hATTR by Emerging therapies in the United States

14.9. EU-5 Market Size

14.9.1. Total Market size of hATTR in Europe

14.9.2. Market Size of hATTR by Current therapies in Europe

14.9.3. Market Size of hATTR by Emerging therapies in Europe

14.10. Japan

14.10.1. Total Market size of hATTR in Japan

14.10.2. Market Size of hATTR by Current therapies in Japan

14.10.3. Market Size of hATTR by Emerging therapies in Japan

15. KOL Views

16. Market Drivers

17. Market Barriers

18. Unmet Needs

19. Market Access and Reimbursement

19.1. US

19.2. Europe

19.3. Japan

19.4. Reimbursement scenario for Orphan drugs in the 7MM

20. Appendix

20.1. Bibliography

20.2. Report Methodology

21. DelveInsight Capabilities

22. Disclaimer

23. About DelveInsight

List of Table

Table 1: Summary of Hereditary Transthyretin Amyloidosis (hATTR) Market, and Epidemiology (2018–2030)

Table 2: Key Events

Table 3: Diagnostic tools for patients presenting with cardiomyopathy

Table 4: Clinical staging of TTR-FAP (based on Coutinho et al)

Table 5: Disease staging based on PND score

Table 6: Portuguese classification system to evaluate the severity of TTR-FAP

Table 7: Treatment of clinical symptoms of transthyretin familial polyneuropathy (TTR-FAP):

Table 8: Symptom management of TTR-FAP (Based on the Coutinho staging)

Table 9: Typical clinical features of later disease (average 4 years post onset; the usual delay in diagnosis)

Table 10: Total Prevalent Population of hATTR in the 7MM (2018–2030)

Table 11: Total Diagnosed Prevalent Population of HATTR in the 7MM (2018–2030)

Table 12: Total Prevalent Population of hATTR in the US (2018–2030)

Table 13: Total Diagnosed Prevalent Population of hATTR in the US (2018–2030)

Table 14: Type-specific Cases of hATTR in the US (2018–2030)

Table 15: Type-specific Cases of hATTR in the US (2018–2030)

Table 16: Stage-specific Cases of FAP in the United States (2018–2030)

Table 17: New York Heart Association (NYHA) Classification of FAC in the United States (2018–2030)

Table 18: Total Prevalent Population of hATTR in EU5 (2018–2030)

Table 19: Total Diagnosed Prevalent Population of hATTR in EU5 (2018–2030)

Table 20: Type-specific Cases of hATTR in EU5 (2018–2030)

Table 21: Stage-specific Cases of FAP in EU5 (2018–2030)

Table 22: New York Heart Association (NYHA) Classification of FAC in EU5 (2018–2030)

Table 23: Total Prevalent Population of hATTR in Japan (2018–2030)

Table 24: Total Diagnosed Prevalent Population of hATTR in the Japan (2018–2030)

Table 25: Type-specific Cases of hATTR in Japan (2018–2030)

Table 26: Stage-specific Cases of FAP in Japan (2018–2030)

Table 27: New York Heart Association (NYHA) Classification of FAC in Japan (2018–2030)

Table 28: Organizations Contributing Toward the Fight Against Hereditary transthyretin amyloidosis (hATTR)

Table 29: List of marketed drugs for hATTR in the 7MM

Table 30: Patents for Onpattro

Table 31: Onpattro, Clinical Trial Description, 2021

Table 32: Tegsedi, Clinical Trial Description, 2021

Table 33: Comparison of emerging drugs under development

Table 34: Vutrisiran, Clinical Trial Description, 2021

Table 35: AKCEA-TTR-LRx, Clinical Trial Description, 2021

Table 36: Acoramidis, Clinical Trial Description, 2021

Table 37: CRX-1008, Clinical Trial Description, 2021

Table 38: PRX004, Clinical Trial Description, 2021

Table 39: NTLA-2001, Clinical Trial Description, 2021

Table 40: Market Size of hATTR in the 7MM in USD Million (2018–2030)

Table 41: Market Size of hATTR by Current therapies in the 7MM in USD Million (2018–2030)

Table 42: Market Size of hATTR by Emerging therapies in the 7MM in USD Million (2018–2030)

Table 43: Key Market Forecast Assumptions for NTLA-2001- hATTR-PN

Table 44: Key Market Forecast Assumptions for NTLA-2001- hATTR- CM

Table 45: Key Market Forecast Assumptions for vutrisiran- hATTR- PN

Table 46: Key Market Forecast Assumptions for vutrisiran- hATTR- CM

Table 47: Key Market Forecast Assumptions for AKCEA-TTR-LRx- hATTR- PN

Table 48: Key Market Forecast Assumptions for AKCEA-TTR-LRx- hATTR- CM

Table 49: Key Market Forecast Assumptions for Acoramidis -hATTR- PN

Table 50: Key Market Forecast Assumptions for Acoramidis- hATTR- CM

Table 51: Key Market Forecast Assumptions for Tolcapone - hATTR- PN

Table 52: Key Market Forecast Assumptions for PRX004-hATTR-CM

Table 53: Market Size of HATTR in the US, USD Million (2018–2030)

Table 54: Market Size of hATTR by Current therapies in the US, USD Million (2018–2030)

Table 55: Market Size of hATTR by Emerging therapies in the US, USD Million (2018–2030)

Table 56: EU5 Market Size of hATTR in USD Million (2018–2030)

Table 57: EU5 Market Size of hATTR by Current therapies in USD Million (2018–2030)

Table 58: EU5 Market Size of hATTR by Emerging therapies in USD Million (2018–2030)

Table 59: Japan Market Size of hATTR in Japan, USD Million (2018–2030)

Table 60: Market Size of hATTR by Current therapies in Japan, USD Million (2018–2030)

Table 61: Market Size of hATTR by Emerging therapies in Japan, USD Million (2018–2030)

List of Figures

Figure 1: Transthyretin amyloid formation and mechanisms of FAP therapies

Figure 2: Pathogenesis of Transthyretin Amyloidosis

Figure 3: Red flag symptoms of hATTR Amyloidosis

Figure 4::Clinical findings indicative of hATTR

Figure 5: Current treatment pathway for patients with Transthyretin Amyloidosis with PN

Figure 6: Total Prevalent Population of hATTR in the 7MM (2018–2030)

Figure 7: Total Diagnosed Prevalent Population of hATTR in the 7MM (2018–2030)

Figure 8: Total Prevalent Population of hATTR in the US (2018–2030)

Figure 9: Total Diagnosed Prevalent Population of hATTR in the US (2018–2030)

Figure 10: Stage-specific Cases of FAP in the United States (2018–2030)

Figure 11: NYHA Classification of FAC in the United States (2018–2030)

Figure 12: Total Prevalent Population of hATTR in EU5 (2018–2030)

Figure 13: Total Diagnosed Prevalent Population of hATTR in EU5 (2018–2030)

Figure 14: Type-specific Cases of hATTR in EU5 (2018–2030)

Figure 15: Stage-specific Cases of FAP in EU5 (2018–2030)

Figure 16: New York Heart Association (NYHA) Classification of FAC in EU5 (2018–2030)

Figure 17: Total Prevalent Population of hATTR in Japan (2018–2030)

Figure 18: Total Diagnosed Prevalent Population of hATTR in Japan (2018–2030)

Figure 19: Type-specific Cases of hATTR in Japan (2018–2030)

Figure 20: Stage-specific Cases of FAP in Japan (2018–2030)

Figure 21: New York Heart Association (NYHA) Classification of FAC in Japan (2018–2030)

Figure 22: Market Size of hATTR in the 7MM in USD Million (2018–2030)

Figure 23: Market Size of hATTR by Current therapies in the 7MM in USD Million (2018–2030)

Figure 24: Market Size of hATTR by Emerging therapies in the 7MM in USD Million (2018–2030)

Figure 25: Market Size of hATTR in the US, USD Millions (2018–2030)

Figure 26: Market Size of hATTR by Current therapies in the US, USD Million (2018–2030)

Figure 27: Market Size of hATTR by Emerging therapies in the US, USD Million (2018–2030)

Figure 28: Market Size of hATTR in EU5, USD Million (2018–2030)

Figure 29: EU5 Market Size of HATTR by Current therapies in USD Million (2018–2030)

Figure 30: EU5 Market Size of hATTR by Emerging therapies in USD Million (2018–2030)

Figure 31: Market Size of hATTR in Japan, USD Million (2018–2030)

Figure 32: Japan Market Size of hATTR by Current Therapies, USD Million (2018–2030)

Figure 33: Japan Market Size of hATTR by Emerging Therapies, USD Million (2018–2030)

Figure 34: Onpattro Approval and Reimbursement Milestones

Pfizer
Alnylam Pharmaceuticals
Akcea Therapeutics
Ionis Pharmaceuticals
AstraZeneca
Eidos Therapeutics
Corino Therapeutics
Prothena
Novo Nordisk
Intellia Therapeutics
Regeneron Pharmaceuticals

 

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