Global Hereditary Amyloidosis Treatment Market to Reach USD 48.2 billion by 2035 with 15.3% CAGR

The global Hereditary Amyloidosis Treatment Market is set to expand rapidly, growing from an estimated USD 11.6 billion in 2025 to USD 48.2 billion by 2035. This remarkable trajectory represents a compound annual growth rate (CAGR) of 15.3%, underscoring the increasing demand for therapies addressing rare hereditary disorders. Growing recognition of amyloid-related genetic conditions, combined with advancements in treatment innovation, is shaping this market into one of the most promising areas in rare disease therapeutics.

Hereditary amyloidosis, a complex and often underdiagnosed condition, is now receiving unprecedented attention. With rising awareness among healthcare providers and patients, the focus is shifting toward targeted, disease-modifying treatments that address the underlying causes rather than merely managing symptoms.

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Key Growth Drivers

The growth of this market is propelled by three primary factors: the rising prevalence of hereditary amyloidosis, increasing investments in research and development, and the availability of targeted therapies. Clinical trials investigating gene-silencing and source treatments are introducing breakthrough options, while improvements in healthcare infrastructure and specialized treatment centers are enhancing patient access to care.

Another crucial factor is the global shift toward precision and personalized medicine. Physicians are increasingly using genetic screening tools to identify at-risk patients earlier, facilitating proactive treatment strategies. As awareness grows, patients are being diagnosed sooner, creating greater demand for therapies that can slow or halt disease progression.

Disease Type Insights: ATTR Amyloidosis Leads

ATTR amyloidosis, the most common hereditary form, is expected to dominate the market, accounting for 64.1% of total revenue in 2025. Its prevalence, coupled with the availability of targeted therapies addressing transthyretin protein misfolding, is fueling its leadership. Genetic screening programs are accelerating diagnosis, while disease-modifying treatments are demonstrating improved outcomes.

Ongoing research into long-term therapeutic safety and effectiveness continues to strengthen clinical confidence. As innovation progresses, ATTR amyloidosis will remain the central focus of drug development, sustaining its dominant market share over the forecast period.

Treatment Type Insights: Source Treatment at the Forefront

Source treatment, which targets the root cause of amyloid protein accumulation, is anticipated to capture 57.6% of total market revenue in 2025. Unlike supportive treatment that only addresses symptoms, source therapies modify disease progression, offering measurable improvements in survival and quality of life.

Patient demand for long-term solutions has accelerated adoption of these approaches. Hospitals and specialized clinics are establishing structured protocols for safe and effective administration, building confidence among healthcare providers. With continuous advancements in delivery methods and formulations, source treatment is expected to maintain leadership throughout the forecast horizon.

End-User Insights: Hospitals as the Primary Point of Care

Hospitals are projected to dominate end-user adoption, capturing 62.8% of market revenue in 2025. This reflects their role as hubs for specialized care, advanced diagnostic services, and administration of complex therapies. Hospitals offer multidisciplinary teams and infrastructure capable of handling rare genetic disorders, making them the natural choice for hereditary amyloidosis patients.

The consolidation of treatment services within hospital networks ensures integrated management, from diagnosis to long-term monitoring. With increased awareness of rare disease care, hospitals will continue to serve as the primary treatment setting in this market.

Market Dynamics and Challenges

While the market outlook is positive, challenges remain. The complexity of hereditary amyloidosis often results in delayed diagnosis, affecting timely intervention. Treatment costs also pose accessibility concerns, particularly in low- and middle-income countries. Moreover, while therapies can slow disease progression, complete recovery is not yet possible.

Nevertheless, manufacturers and healthcare providers are addressing these challenges by expanding reimbursement policies, increasing clinical research, and developing more affordable options. The ongoing wave of innovation ensures that limitations are progressively being mitigated.

Regional Outlook

North America remains the largest regional market, driven by advanced healthcare infrastructure, early adoption of novel therapies, and strong reimbursement frameworks. The U.S. market alone is projected to grow from USD 4.3 billion in 2025 to USD 14.7 billion by 2035, with a CAGR of 13.0%.

Europe is also expanding steadily, supported by government initiatives funding rare disease research. Germany, in particular, is expected to post a CAGR of 17.6%, solidifying its leadership within the region.

Asia-Pacific is emerging as the fastest-growing region, led by China with a forecast CAGR of 20.7% and India at 19.2%. Improvements in healthcare access and subsidized medicines are enabling rapid adoption of hereditary amyloidosis treatments. Japan and South Korea are also notable growth contributors, with the markets valued at USD 544.9 million and USD 307.4 million, respectively, in 2025.

Latin America and the Middle East & Africa, though smaller in scale, are witnessing steady progress as awareness of hereditary conditions and supportive care infrastructure improves.

Competitive Landscape

The market is shaped by a combination of established pharmaceutical leaders and emerging biotechnology innovators. Companies such as Alnylam Pharmaceuticals, Pfizer, Ionis Pharmaceuticals, Prothena Corporation, Akcea Therapeutics, and Eidos Therapeutics remain at the forefront, driving advancements in both source and supportive therapies.

Meanwhile, new entrants and niche biotech firms are actively pursuing clinical trials, contributing to a competitive and dynamic landscape. Collaborations, licensing agreements, and research partnerships are common strategies as companies aim to broaden their product portfolios and accelerate commercialization. This mix of established giants and agile newcomers ensures continuous innovation and wider access for patients.

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Market Outlook and Future Trends

Looking ahead, the Hereditary Amyloidosis Treatment Market is expected to evolve with several defining trends. Gene-silencing therapies are gaining momentum, with increasing clinical validation and regulatory support. Advances in precision medicine and personalized care are reshaping treatment protocols, ensuring that therapies are tailored to patient-specific genetic profiles.

Digital health technologies, including remote monitoring and biomarker-based diagnostic tools, will also play a role in enabling early detection and treatment adherence. Combined with global efforts to strengthen healthcare infrastructure for rare diseases, these innovations set the stage for long-term growth.

The next decade is likely to witness not only expanded adoption of current therapies but also the introduction of new modalities capable of altering disease trajectories more effectively than ever before.

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