Pharmaceuticals

The Rising Focus On Gene Therapy Is Fueling The Growth Of The Market Due To Its Ability To Target And Correct Defective Genes For Lasting Familial Amyloid Polyneuropathy Treatment Is Supporting Development Across The Familial Amyloid Polyneuropathy Market

May 19, 2026 by Marketing Posting

The Business Research Company’s 2026 market reports feature advanced enhancements such as market attractiveness analysis, total addressable market evaluation, company benchmarking matrices, interactive Excel dashboards, expanded supply chain intelligence, emerging startup coverage, and detailed product insights, delivering more actionable and strategically valuable research.

What Is The Estimated Growth In Market Size Of The Familial Amyloid Polyneuropathy Market During 2026–2030?

The market size for familial amyloid polyneuropathy has demonstrated robust expansion over the past few years. Looking ahead, its valuation is projected to increase from $1.65 billion in 2025 to $1.79 billion in 2026, advancing at a compound annual growth rate (CAGR) of 8.3%. Factors contributing to this historical expansion include advancements in rare disease diagnosis, heightened genetic disorder awareness, progress in transplant medicine development, intensified neurology research, and the broader adoption of supportive care.

The familial amyloid polyneuropathy market is projected to experience substantial expansion in the coming years. Its valuation is anticipated to reach $2.43 billion by 2030, exhibiting a compound annual growth rate (CAGR) of 8.0%. This projected growth stems from factors such as advancements in gene therapy, widespread early genetic testing, increased funding for rare diseases, the adoption of precision medicine, and the expansion of specialty pharmacies. Key developments expected during this period include the wider acceptance of TTR stabilizers, the proliferation of gene-silencing treatments, enhanced genetic screening practices, a heightened focus on therapies for rare conditions, and the establishment of more specialty care centers.

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What Key Growth Drivers Are Influencing The Familial Amyloid Polyneuropathy Market?

An increasing emphasis on gene therapy is anticipated to boost the familial amyloid polyneuropathy market’s expansion in the future. Gene therapy, a therapeutic approach, works by altering or substituting faulty genes to tackle the root cause of illnesses. This treatment is gaining traction because it aims to correct defective genes, providing enduring or permanent remedies instead of merely alleviating symptoms. Specifically for familial amyloid polyneuropathy, gene therapy focuses on rectifying or deactivating the flawed transthyretin (TTR) gene, thereby inhibiting the formation of detrimental amyloid protein. Such intervention assists in decreasing amyloid deposits, thereby decelerating the disease’s progression and safeguarding nerve function. As an illustration of this trend, data from July 2023, jointly published by the American Society of Gene & Cell Therapy (ASGCT), a US-based professional membership organization, and Citeline, a US-based provider of drugs and devices, indicated that 247 gene therapies were in Phase II at the close of the first quarter of 2023; this figure subsequently increased by 5% to reach 260 by the end of the second quarter. Consequently, the heightened attention on gene therapy is spurring the expansion of the familial amyloid polyneuropathy market.

What Key Segment Areas Are Included In The Familial Amyloid Polyneuropathy Market Assessment?

The familial amyloid polyneuropathy market covered in this report is segmented –

1) By Treatment: Medication, Gene Therapy, Supportive Care, Pain Management, Symptomatic Treatment

2) By Diagnosis: Genetic Testing, Biopsy, Imaging, Other Diagnosis

3) By Patient Age Group: Pediatric, Adult, Geriatric

4) By Distribution Channel: Hospital Pharmacies, Retail Pharmacies, Online Pharmacies, Specialty Pharmacies, Direct Sales

5) By End-User: Hospitals, Specialty Clinics, Research Institutes, Other End-Users

Subsegments:

1) By Medication: Transthyretin (TTR) Stabilizers, Transthyretin (TTR) Gene Silencers, Tafamidis, Diflunisal, Patisiran

2) By Gene Therapy: Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Or Cas9-Based Therapy, RNA Interference (RNAi) Therapy, Antisense Oligonucleotides (ASO) Therapy

3) By Supportive Care: Physical Therapy, Occupational Therapy, Nutritional Support, Psychological Counseling

4) By Pain Management: Non-Steroidal Anti-Inflammatory Drugs (NSAIDs), Opioids, Antidepressants, Anticonvulsants

5) By Symptomatic Treatment: Gastrointestinal Symptom Management, Cardiovascular Symptom Management, Autonomic Dysfunction Management, Orthostatic Hypotension Treatment

Which Trends Are Shaping The Familial Amyloid Polyneuropathy Market Landscape?

Leading companies within the familial amyloid polyneuropathy market are concentrating on developing advanced treatment solutions, such as RNA interference (RNAi) therapeutics, with the goal of slowing disease progression and improving patient outcomes. RNA interference (RNAi) therapeutics are sophisticated treatments designed to silence disease-causing genes by targeting their messenger RNA (mRNA), effectively blocking the production of harmful proteins and addressing genetic diseases at their source. For example, in March 2025, Alnylam Pharmaceuticals, a US-based biopharmaceutical company, launched AMVUTTRA (vutrisiran) following its approval by the 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, this treatment is designed to address both ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN). By reducing the production of the transthyretin (TTR) protein, which is the underlying cause of the disease, AMVUTTRA significantly lowers cardiovascular mortality, hospitalizations, and urgent heart failure visits, a finding demonstrated in the HELIOS-B Phase 3 clinical trial.

Which Companies Are Influencing The Familial Amyloid Polyneuropathy Market?

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

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Which Geographic Region Represents The Largest Familial Amyloid Polyneuropathy Market Share?

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.

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