U.S. Transthyretin Amyloidosis Treatment Market is Driven by Increasing R&D Investments and New Product Launches
The U.S. transthyretin amyloidosis treatment market is estimated to be valued at USD 59.7 Mn in 2024, exhibiting a CAGR of 9.4% over the forecast period (2024-2031). The market is witnessing high growth due to increasing R&D investments by key players for the development of novel treatment therapies and a growing number of clinical trials for rare disease treatment. Furthermore, recent approvals and launches of new drugs to treat different types of transthyretin amyloidosis in the country are further fueling the market growth.
Market Dynamics:
The growth of the U.S. transthyretin amyloidosis treatment market is driven by two key factors. Firstly, increasing R&D investments by pharmaceutical companies to develop innovative treatment therapies for transthyretin amyloidosis is expected to boost market growth during the forecast period. Several drug makers are conducting clinical trials to evaluate drug candidates for different hereditary and wild-type forms of the disease which will drive market revenues. Secondly, the approval and launch of new drugs by the U.S. Food and Drug Administration in recent times has enhanced treatment options available to physicians and patients.
Increasing Prevalence of Transthyretin Amyloidosis Diseases
One of the major drivers for the U.S. transthyretin amyloidosis treatment market is the increasing prevalence of transthyretin amyloidosis diseases in the country. Transthyretin amyloidosis is a rare, progressive disease caused by mutations in the transthyretin (TTR) gene. These genetic mutations cause abnormal deposits of TTR amyloid proteins to build up in the body's organs and tissues. According to the Transthyretin Amyloidosis Supporters, an estimated 50,000 Americans are affected by transthyretin amyloidosis. The rising disease burden is expected to drive greater demand for diagnostic tests, treatments, and disease management services in the U.S.
Advancements in Treatment and Diagnostic Options
Another key market driver is the ongoing advancements being made in treatment and diagnostic options for transthyretin amyloidosis. Previously, treatment options for transthyretin amyloidosis were very limited. However, in recent years several new drugs have been approved by the U.S. FDA that can halt or slow the progression of the disease. These include tafamidis, inotersen, and patisiran. In addition, novel diagnostic techniques using scintigraphy and genetic testing have improved early detection rates. The introduction of newer and more effective treatment modalities is expanding the addressable patient pool and driving higher spending on transthyretin amyloidosis management.
High Treatment Costs
One major restraint on the U.S. transthyretin amyloidosis treatment market is the extremely high costs associated with transthyretin amyloidosis treatment. Most newly approved drugs have price tags in the six-figure range per year. For example, tafamidis was priced at US$ 225,000 for the first year and US$ 100,000 for subsequent years when it launched. Such high costs place significant financial burden on patients and payers. While some patients may be able to access treatment through assistance programs or subsidies, the overall high drug prices limit broader market access and uptake of new therapies.
Lack of Disease Awareness
Another market barrier is the lack of awareness about transthyretin amyloidosis, both within the medical community and among the general public. Due to the rarity of the disease, many primary care physicians and neurologists have limited knowledge about its symptoms and diagnostic criteria. Furthermore, patients themselves are often misdiagnosed before receiving a definitive transthyretin amyloidosis confirmation. The low disease awareness contributes to diagnostic delays and late treatment initiation. Concerted awareness campaigns are needed to educate healthcare providers and empower early patient identification.
Increased Research Funding
A major market opportunity lies in the increased research funding flowing into transthyretin amyloidosis. Both government agencies and private sector organizations are ramping up support for research aimed at developing novel treatment targets, earlier diagnostics, and disease-modifying therapies. For example, the transthyretin amyloidosis supporters received a US$ 25 million grant in 2021 for a research consortium. Greater investments into transthyretin amyloidosis clinical trials and research infrastructure will help catalyze the development of advanced solutions in the coming years.
Partnerships with Health Systems
Partnering with large healthcare systems provides another avenue for market growth. Hospitals and clinics play a central role in diagnosing and managing transthyretin amyloidosis patients. Biopharmaceutical companies can collaborate with health systems to raise awareness amongst referring physicians, train specialists, implement early detection programs, and support care coordination. Such partnerships will increase the customer reach of new therapies while also improving long-term treatment access and outcomes for patients.
Key Developments
- On October 9, 2024, Alnylam Pharmaceuticals, Inc., the RNAi therapeutics company, announced the submission of its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for vutrisiran, an investigational RNAi therapeutic in the development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM). Vutrisiran is the generic name for AMVUTTRA, which is currently approved by the U.S. FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.
- On February 5, 2024, BridgeBio Pharma, Inc., a pharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the company’s New Drug Application (NDA) for acoramidis, an investigational drug for the treatment of ATTR-CM
- In March 2023, AstraZeneca, a global biopharmaceutical company, announced the results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The trials showed that eplontersen met its co-primary endpoints through 66 weeks.
Key Players
Alnylam Pharmaceuticals, Inc., Pfizer, Inc., Prothena Corporation Plc, GSK plc, Ionis Pharmaceuticals, Inc., SOM Innovation Biotech, S.L., Eidos Therapeutics, BridgeBio Pharma, Inc., Bristol-Myers Squibb Company, AstraZeneca, Acrotech Biopharma, and Attralus, Inc.
