Familial Amyloid Polyneuropathy Global Market Report 2025

The familial amyloid polyneuropathy market size has grown strongly in recent years. It will grow from $1.52 billion in 2024 to $1.65 billion in 2025 at a compound annual growth rate (CAGR) of 8.4%. The growth during the historic period can be attributed to the rising prevalence of familial amyloid polyneuropathy, improved awareness and diagnosis rates driven by advances in genetic testing, increased adoption of new therapeutic and targeted treatment options, greater investment in research and development for amyloidosis therapies, and a growing elderly population vulnerable to amyloid-related neuropathies.

The familial amyloid polyneuropathy market size is expected to see strong growth in the next few years. It will grow to $2.25 billion in 2029 at a compound annual growth rate (CAGR) of 8.1%. The growth expected during the forecast period can be attributed to expanding government initiatives and funding dedicated to rare disease treatments, a rising number of clinical trials testing innovative therapies, increased patient advocacy and support groups fueling demand for improved treatments, enhanced access to healthcare infrastructure in emerging markets, and greater availability of advanced diagnostic technologies. Key trends anticipated in this period include progress in genetic sequencing technologies, the adoption of personalized medicine, advancements in biomarker identification techniques, improvements in drug delivery systems, and the incorporation of digital health tools and telemedicine.

The increasing emphasis on gene therapy is expected to drive the growth of the familial amyloid polyneuropathy market going forward. Gene therapy involves modifying or replacing faulty genes to treat the root cause of diseases. This approach is gaining traction because it targets and repairs defective genes, potentially providing lasting or permanent cures rather than just alleviating symptoms. For familial amyloid polyneuropathy, gene therapy aims to correct or silence the mutated transthyretin (TTR) gene to prevent the buildup of harmful amyloid proteins. This reduces amyloid deposits, slows disease progression, and helps preserve nerve function. For example, in July 2023, the American Society of Gene & Cell Therapy (ASGCT), a U.S.-based professional organization, and Citeline, a U.S.-based drug and device provider, reported that the number of gene therapies in Phase II clinical trials increased from 247 at the end of the first quarter of 2023 to 260 by the end of the second quarter, a 5% rise. Thus, the growing focus on gene therapy is fueling the expansion of the familial amyloid polyneuropathy market.

Leading companies in the familial amyloid polyneuropathy market are concentrating on developing advanced therapies such as RNA interference (RNAi) treatments to slow disease progression and enhance patient outcomes. RNAi therapeutics work by silencing disease-causing genes through targeting their messenger RNA (mRNA), thereby preventing the production of harmful proteins and addressing genetic disorders at their source. For example, in March 2025, Alnylam Pharmaceuticals, a U.S.-based biopharmaceutical firm, launched AMVUTTRA (vutrisiran), which received FDA approval for the treatment of cardiomyopathy caused by wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults. The therapy is administered as four subcutaneous doses annually and is designed to treat both ATTR-CM and hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN). By reducing transthyretin (TTR) protein production - the underlying cause of the disease - it significantly decreases cardiovascular mortality, hospitalizations, and urgent heart failure visits, as evidenced in the HELIOS-B Phase 3 clinical trial.

In March 2023, Alnylam Pharmaceuticals, a U.S.-based developer of RNAi therapies for hereditary transthyretin-mediated amyloidosis, formed a partnership with Medison Pharma to expand global access to innovative RNAi treatments. The collaboration seeks to speed up and widen patient availability of Alnylam’s RNAi therapeutics for rare and serious diseases by utilizing Medison Pharma’s integrated commercialization platform across multiple international markets. Medison Pharma is a pharmaceutical company headquartered in Israel.

Major players in the familial amyloid polyneuropathy market are Pfizer Inc., AstraZeneca plc, Novo Nordisk A/S, Regeneron Pharmaceuticals Inc., NYU Langone Hospitals, Alexion Pharmaceuticals Inc., Alnylam Pharmaceuticals Inc., Mount Sinai Medical Center, PTC Therapeutics Inc., Ionis Pharmaceuticals Inc., Mayo Foundation for Medical Education and Research, Arcturus Therapeutics Inc., Prothena Corporation plc, Precision BioSciences Inc., Intellia Therapeutics Inc., Creative Biolabs, Stanford Health Care, Attralus Inc., Duke University Health System, and YolTech Therapeutics.

North America was the largest region in the familial amyloid polyneuropathy market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in familial amyloid polyneuropathy report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the familial amyloid polyneuropathy market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Familial amyloid polyneuropathy (FAP) is a rare inherited disorder caused by mutations in the transthyretin (TTR) gene, leading to the buildup of amyloid proteins in peripheral nerves and vital organs. This progressive condition primarily affects the nervous system, causing sensory, motor, and autonomic impairments. Early diagnosis and proper management focus on slowing disease progression, easing symptoms, and improving the patient’s quality of life.

The main treatment options for familial amyloid polyneuropathy include medication, gene therapy, supportive care, pain management, and symptomatic relief. Medications help by slowing the disease’s advancement, reducing symptoms, and enhancing patients’ quality of life. Diagnosis can be performed using various techniques such as genetic testing, biopsy, imaging, and others, across different age groups including pediatric, adult, and geriatric patients. Treatments are distributed through multiple channels like hospital pharmacies, retail and online pharmacies, specialty pharmacies, and direct sales, and are utilized by various end-users including hospitals, specialty clinics, research institutes, and more.

The familial amyloid polyneuropathy market research report is one of a series of new reports that provides familial amyloid polyneuropathy market statistics, including the familial amyloid polyneuropathy industry global market size, regional shares, competitors with the familial amyloid polyneuropathy market share, detailed familial amyloid polyneuropathy market segments, market trends, opportunities, and any further data you may need to thrive in the familial amyloid polyneuropathy industry. This familial amyloid polyneuropathy market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

The familial amyloid polyneuropathy market consists of revenues earned by entities providing services such as disease monitoring, patient support and education, neurological assessment, and genetic counseling. The market value includes the value of related goods sold by the service provider or included within the service offering. The familial amyloid polyneuropathy market also includes sales of transthyretin stabilizers, diagnostic kits, and monitoring equipment. Values in this market are ‘factory gate’ values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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