Familial Amyloid Polyneuropathy Market Report 2026

The familial amyloid polyneuropathy market size has grown strongly in recent years. It will grow from $1.65 billion in 2025 to $1.79 billion in 2026 at a compound annual growth rate (CAGR) of 8.3%. The growth in the historic period can be attributed to rare disease diagnosis, genetic disorder awareness, transplant medicine development, neurology research, supportive care adoption.

The familial amyloid polyneuropathy market size is expected to see strong growth in the next few years. It will grow to $2.43 billion in 2030 at a compound annual growth rate (CAGR) of 8%. The growth in the forecast period can be attributed to gene therapy innovation, early genetic testing, rare disease funding, precision medicine adoption, specialty pharmacy growth. Major trends in the forecast period include growing adoption of ttr stabilizers, expansion of gene silencing therapies, increased genetic screening, rising focus on rare disease treatment, growth of specialty care centers.

The rising focus on gene therapy is expected to propel the growth of the familial amyloid polyneuropathy (FAP) market in the coming years. Gene therapy is a treatment approach that modifies or replaces defective genes to address the root cause of diseases. Its popularity is increasing because it targets faulty genes directly, offering lasting or potentially permanent cures rather than merely alleviating symptoms. Gene therapy for familial amyloid polyneuropathy focuses on correcting or silencing the defective transthyretin (TTR) gene to prevent harmful amyloid protein formation. This intervention helps reduce amyloid deposits, slow disease progression, and preserve nerve function. For instance, in July 2023, according to the American Society of Gene & Cell Therapy (ASGCT), a US-based professional membership organization, and Citeline, a US-based provider of drugs and devices, there were 247 gene therapies in Phase II at the end of Q1 2023, which rose by 5% to 260 by the end of Q2 2023. Therefore, the increasing focus on gene therapy is driving the growth of the familial amyloid polyneuropathy market.

Major companies operating in the familial amyloid polyneuropathy market are developing advanced treatment solutions, such as RNA interference (RNAi) therapeutics, to slow disease progression and improve patient outcomes. RNAi therapeutics are designed to silence disease-causing genes by targeting their messenger RNA (mRNA), blocking the production of harmful proteins and addressing genetic diseases at their source. For instance, in March 2025, Alnylam Pharmaceuticals, a US-based biopharmaceutical company, launched AMVUTTRA (vutrisiran), which received approval from the U.S. Food and Drug Administration (FDA) for treating cardiomyopathy caused by wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults. Administered as four subcutaneous doses per year, the therapy is designed to treat both ATTR-CM and the polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR-PN). By reducing production of transthyretin (TTR) protein - the root cause of the disease - it significantly lowers cardiovascular mortality, hospitalizations, and urgent heart failure visits, as demonstrated in the HELIOS-B Phase 3 clinical trial.

In March 2023, Alnylam Pharmaceuticals partnered with Medison Pharma to expand global access to innovative RNAi therapies. The collaboration aims to accelerate and broaden patient access to Alnylam’s RNAi therapeutics for rare and severe diseases by leveraging Medison Pharma’s unified international commercialization platform. Medison Pharma is an Israel-based pharmaceutical company.

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

North America was the largest region in the familial amyloid polyneuropathy market in 2025. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the familial amyloid polyneuropathy market report are Asia-Pacific, South East Asia, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the familial amyloid polyneuropathy market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Taiwan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Tariffs have influenced the familial amyloid polyneuropathy market by increasing the cost of gene therapies, RNA-based drugs, and specialized diagnostic kits. Medication and genetic testing segments are most affected, especially in North America and Europe due to reliance on imported advanced therapeutics. Specialty pharmacies and hospitals face higher treatment costs. However, tariffs are encouraging domestic biopharmaceutical production and rare-disease drug manufacturing.

The familial amyloid polyneuropathy market research report is one of a series of new reports that provides familial amyloid polyneuropathy market statistics, including familial amyloid polyneuropathy industry global market size, regional shares, competitors with a familial amyloid polyneuropathy market share, detailed familial amyloid polyneuropathy market segments, market trends and 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 scenario of the industry.

Familial amyloid polyneuropathy (FAP) is a rare genetic 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, resulting in sensory, motor, and autonomic dysfunction. Early diagnosis and effective management focus on slowing disease progression, relieving symptoms, and improving quality of life.

The main types of FAP treatment include medication, gene therapy, supportive care, pain management, and symptomatic treatment. Medications help slow disease progression, alleviate symptoms, and enhance patient well-being. Diagnosis involves methods such as genetic testing, biopsy, and imaging, and patients may be pediatric, adult, or geriatric. Treatments are distributed through hospital pharmacies, retail pharmacies, online pharmacies, specialty pharmacies, and direct sales, serving end-users including hospitals, specialty clinics, research institutes, and others.

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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