Familial Amyloid Polyneuropathy (FAP) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034

Familial Amyloid Polyneuropathy (FAP) Market Outlook

Thelansis’s “Familial Amyloid Polyneuropathy (FAP) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034" 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).

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.

 

Geography coverage:

G8 (United States, EU5 [France, Germany, Italy, Spain, U.K.], Japan, and China)

Insights driven by robust research, including:

• In-depth interviews with leading KOLs and payers
• Physician surveys
• RWE analysis for claims and EHR datasets
• Secondary research (e.g., peer-reviewed journal articles, third-party research databases)

Deliverables format and updates*:

• Detailed Report (PDF)
• Market Forecast Model (MS Excel-based automated dashboard)
• Epidemiology (MS Excel; interactive tool)
• Executive Insights (PowerPoint presentation)
• Others: regular updates, customizations, consultant support

*As per Thelansis’s policy, we ensure that we include all the recent updates before releasing the report content and market model.

Salient features of Market Forecast model:

• 10-year market forecast (2024–2034)
• Bottom-up patient-based market forecasts validated through the top-down sales methodology
• Covers clinically and commercially-relevant patient populations/ line of therapies
• Annualized drug-level sales and patient share projections
• Utilizes our proprietary Epilansis and Analog tool (e.g., drug uptake and erosion) datasets and conjoint analysis approach
• Detailed methodology/sources & assumptions
• Graphical and tabular outputs
• Users can customize the model based on requirements

Key business questions answered:

• How can drug development and lifecycle management strategies be optimized across G8 markets (US, EU5, Japan, and China)?
• How large is the patient population in terms of incidence, prevalence, segments, and those receiving drug treatments?
• What is the 10-year market outlook for sales and patient share?
• Which events will have the greatest impact on the market’s trajectory?
• What insights do interviewed experts provide on current and emerging treatments?
• Which pipeline products show the most promise, and what is their potential for launch and future positioning?
• What are the key unmet needs and KOL expectations for target profiles?
• What key regulatory and payer requirements must be met to secure drug approval and favorable market access?
• and more…

Read more: Familial Amyloid Polyneuropathy (FAP) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034