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

Systemic Amyloidosis Market Outlook

Thelansis’s “Systemic 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 Systemic Amyloidosis treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Systemic Amyloidosis Overview

Systemic amyloidosis (SA) encompasses a group of rare disorders characterized by the deposition of misfolded protein aggregates as insoluble fibrils within tissues, resulting in progressive organ dysfunction, disability, and potentially fatal outcomes. Fourteen proteins have been identified as potential etiological agents of SA, with precise identification crucial for targeted therapy specific to each disease subtype. The second most prevalent subtype is transthyretin amyloid protein (ATTR) amyloidosis, which includes both variant ATTR (ATTRv) and wild-type ATTR (ATTRwt). Other less common subtypes include serum amyloid A (AA) amyloidosis, whose incidence is declining due to improved treatment of underlying chronic inflammatory conditions and other hereditary and acquired forms. SA predominantly affects the heart, kidneys, and peripheral and autonomic nervous systems but can also involve other organs, leading to a broad spectrum of clinical syndromes. The nonspecific nature of its manifestations often complicates the diagnostic process, resulting in prolonged patient journeys marked by numerous medical consultations, examinations, misdiagnoses, and delayed definitive diagnosis. Despite advancements in the diagnosis and treatment of amyloidosis, it remains a condition associated with significant morbidity and high early mortality rates, particularly in patients with cardiac involvement. Early disease recognition and timely intervention are critical to prevent further organ damage.

Systemic amyloidosis is classified based on the chemical composition of the misfolded protein. The amyloid comprises two components:

1. A constant unit, most commonly the amyloid P component.
2. A variable unit, such as:

• Immunoglobulin light chain (producing 'AL' amyloid)
• Transthyretin (producing ATTR amyloid)
• Beta-2 microglobulin (Aβ2M) leads to systemic amyloidosis in chronic dialysis.

Cutaneous manifestations vary depending on the site of amyloid deposition and the degree of local tissue destruction, occurring in 30–40% of patients with primary systemic AL amyloidosis. Complications are variable and depend on the site and extent of involvement:

• Skin, hair, and nail involvement can lead to disfigurement and psychological distress.
• Macroglossia can cause chewing and swallowing difficulties.
• Organ failure and death, particularly due to renal failure, nephrotic syndrome, and cardiomyopathy.

Conventional high-dose plasma cell-directed chemotherapy with melphalan and dexamethasone, sometimes followed by autologous stem cell transplant in those well enough to tolerate it, is a common treatment approach.

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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