Transthyretin Amyloidosis – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Transthyretin Amyloidosis Market Outlook

Thelansis’s “Transthyretin Amyloidosis Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Transthyretin Amyloidosis treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Transthyretin Amyloidosis Overview

Amyloidosis is a systemic infiltrative disease characterized by depositing insoluble proteins outside cells. Transthyretin Amyloidosis occurs when a toxic substance called amyloid is deposited in the extracellular space of the heart. Amyloid consists of abnormally folded proteins and other matrix-forming components such as proteoglycans, glycosaminoglycans, collagen, and laminin. These misfolded proteins originate from amyloid light (AL) chain proteins and amyloid transthyretin (ATTR). It may manifest primarily or be discovered incidentally in patients with other signs and symptoms of systemic amyloidosis. Following are the various etiological types of Transthyretin Amyloidosis:

1. Primary amyloidosis (also known as amyloid light chain amyloidosis or AL amyloidosis) is caused by the deposition of AL fibrils produced by abnormal plasma cells in individuals with plasma cell dyscrasia, such as multiple myeloma.
2. Secondary amyloidosis (AA amyloidosis) is caused by the deposition of serum amyloid A, an inflammatory protein produced in chronic inflammation conditions.
3. Senile systemic amyloidosis (also known as wild transthyretin or ATTRw) is caused by age-related amyloid deposition, which consists of normal transport-thyroxine-and-retinol (TTR) protein. This is the most common type of Transthyretin Amyloidosis.
4. Mutant TTR causes familial amyloidosis (ATTRm).
5. Isolated atrial amyloidosis is caused by the deposition of amyloid derived from the atrial natriuretic peptide.

Deposing amyloid in the heart can lead to various heart diseases through multiple mechanisms. Direct infiltration of the interstitial space increases the ventricular walls' thickness and stiffness, leading to ventricular diastolic dysfunction. In AL amyloidosis, amyloid deposition in arterioles can cause angina or, rarely, myocardial infarction. Amyloid infiltration in the atria can induce structural changes, providing a substrate for atrial fibrillation. The prognosis varies depending on the specific type of Transthyretin Amyloidosis. The average survival time in untreated patients is as follows: AL (primary) amyloidosis ranges from 9 to 24 months, ATTR familial amyloidosis from 7 to 10 years, senile amyloidosis from 5 to 7 years, and AA (secondary) amyloidosis is more than ten years.

• The prevalence of Transthyretin Amyloidosis is higher among men (70 per 100,000 person-years) than women (44 per 100,000 person-years). The incidence is similar among men (18 per 100,000 person-years) and women (15 per 100,000 person-years).

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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