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

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.

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

KOLs insights of Familial Amyloid Polyneuropathy (FAP) across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.
Familial Amyloid Polyneuropathy (FAP) Market Forecast Patient Based Forecast Model (MS. Excel Based Automated Dashboard), which 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.
Thelansis Competitive Intelligence (CI) practice has been established based on a deep understanding of the pharma/biotech business environment to provide an optimized support system to all levels of the decision-making process. It enables business leaders in forward-thinking and proactive decision-making. Thelansis supports scientific and commercial teams in seamless CI support by creating an AI/ ML-based technology-driven platform that manages the data flow from primary and secondary sources.
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