Familial Amyloid Polyneuropathy (FAP) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Familial Amyloid Polyneuropathy (FAP) Market Outlook

Thelansis’s “Familial Amyloid Polyneuropathy (FAP) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" 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 Familial Amyloid Polyneuropathy treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Familial Amyloid Polyneuropathy (FAP) Overview

Familial amyloid polyneuropathy (FAP) is a neurodegenerative condition characterized by the abnormal accumulation of mutant transthyretin (TTR) amyloid fibrils outside of cells, primarily in the peripheral nervous system. Mutations in the TTR gene lead to the destabilization of the TTR protein, causing it to change from its normal tetrameric form into a form that can form amyloid fibrils. In countries where FAP is prevalent, it typically manifests as small fiber neuropathy that varies in severity based on the length of the affected nerves. There are several distinct types of FAP, such as the Portuguese, Japanese, and Swedish variants. While they share similar clinical characteristics, they differ in the age at which symptoms typically appear. For instance, Portuguese FAP can begin in a person's third or fourth decade, Japanese FAP typically emerges between ages 25 and 35, and Swedish FAP tends to start after age 55. Despite these differences, all three types share the underlying biochemical problem of producing abnormal or mutant forms of transthyretin. Therefore, they are collectively referred to as ATTR (familial ATTR). Transthyretin, previously known as thyroxin-binding pre-albumin, is a protein composed of four identical subunits and is a carrier for thyroxin and retinol-binding protein. Multiple amino acid substitutions in transthyretin can lead to the development of FAP, but the most common substitution is replacing valine with methionine at position 30 (Met-Val 30). This mutation is associated with FAP type 1 or the Portuguese, Japanese, and Swedish. In addition to transthyretin mutations, FAP can also rarely result from mutations in other proteins, including apolipoprotein A-1 (a high-density lipoprotein), fibrinogen A γ, lysozyme, and gelsolin (a cytoplasmic protein involved in actin filament interactions). The clinical symptoms of FAP type 1 ATTR neuropathy closely resemble those of AL amyloid neuropathy, with common signs of sensorimotor and autonomic neuropathy. FAP type 2, the Indiana type, is typically associated with transthyretin mutations at Ser84 or His58. Patients with this type often present with carpal tunnel syndrome and vitreous opacities, and their sensorimotor and autonomic neuropathies tend to be relatively mild. FAP type 3, or the Iowa type, is linked to an abnormal apolipoprotein A-1 and shares many similarities with FAP type 1. However, carpal tunnel syndrome is less frequently observed, and autonomic neuropathy is less pronounced. FAP type 4, the Finnish type, results from an abnormal gelsolin protein and manifests as a unique clinical syndrome featuring progressive cranial neuropathies and a distinctive thickening of facial skin. Sensorimotor and autonomic neuropathies associated with this type are typically mild. Liver transplantation (LT) is no longer considered the primary treatment for FAP, as its main goal is to remove the significant source of amyloidogenic TTR. LT can effectively stop disease progression by eliminating 95% of circulating TTR.

• FAP, endemic in Portugal, Sweden, and specific regions of Japan, has been reported in 36 countries worldwide, with an estimated global prevalence ranging from 5,000 to 10,000 individuals.

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: 2021-2032

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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