The global amyloidosis therapeutics market is witnessing a period of transformation, propelled by advancements in rare disease diagnosis, increased research investments, and an expanding pipeline of targeted therapies. Once overlooked due to its rarity, amyloidosis is gaining attention in the medical and pharmaceutical industries as awareness rises and healthcare systems become better equipped to detect and manage complex conditions.

According to market research, the amyloidosis therapeutics industry was valued at US$ 2.7 Bn in 2024, and is projected to expand at a CAGR of 8.2% from 2025 to 2035, crossing US$ 6.3 Bn by the end of 2035.

Understanding Amyloidosis and Its Therapeutic Landscape

Amyloidosis is a group of rare but serious diseases caused by the abnormal deposition of amyloid proteins in tissues and organs, resulting in organ dysfunction. Among its many forms, light-chain (AL) amyloidosis is the most common, often linked to plasma cell disorders like multiple myeloma. Other types include AA amyloidosis, familial transthyretin-related (ATTR) amyloidosis, and senile systemic amyloidosis.

This heterogeneous disease poses diagnostic and therapeutic challenges. However, with rising awareness, more accurate diagnostic tools, and a growing focus on personalized medicine, the therapeutic landscape is evolving rapidly.

Key Drivers Behind Market Growth

✅ 1. Rising Prevalence of Amyloidosis

Previously underdiagnosed, amyloidosis cases are now being more readily identified across global populations, thanks to advancements in diagnostic imaging, biomarkers, and awareness among clinicians. For example, AL amyloidosis, which accounts for nearly 70% of systemic cases, affects an estimated 4,000 people annually in the U.S. alone, primarily between the ages of 50 and 65.

As early diagnosis improves, so does the need for timely and effective treatments, leading to increased demand for therapeutic innovation and market expansion.

✅ 2. Accelerated R&D and Strategic Investments

The rise in research funding and industry collaboration has opened up new treatment pathways. Pharmaceutical giants and biotech startups are investing heavily in RNA interference (RNAi) therapies, monoclonal antibodies, and small-molecule stabilizers.

Notably:

• Intellia Therapeutics reported promising Phase 1 data in 2024 for its gene-editing candidate targeting ATTR amyloidosis.
• Alnylam Pharmaceuticals submitted a supplemental New Drug Application (sNDA) to the FDA for vutrisiran, an RNAi therapy targeting ATTR-CM.

These developments highlight how R&D is not only addressing unmet medical needs but also unlocking commercial potential in this niche segment.

Spotlight on Market Segments

🔬 Monoclonal Antibodies Lead the Therapeutic Space

Monoclonal antibodies (mAbs) dominate the drug type segment due to their precision in targeting amyloid fibrils and minimizing off-target effects. Their disease-modifying potential offers hope for long-term control, and recent approvals reinforce their pivotal role.

For example, in 2023, LEQEMBI (lecanemab), a humanized anti-amyloid-beta antibody by Eisai Co., Ltd. and Biogen Inc., was approved in Japan to slow progression of Alzheimer’s-related amyloidosis, opening new doors for similar approaches in systemic types.

🧬 AL Amyloidosis: The Largest Application Segment

As the most prevalent and life-threatening form of systemic amyloidosis, AL amyloidosis accounts for the largest market share. With high mortality rates if untreated, early intervention with targeted drugs is crucial. Improved therapies for plasma cell disorders (e.g., proteasome inhibitors and immunomodulators) have also had a positive spillover effect on treatment options for AL amyloidosis.

Regional Insights: North America Leading the Way

North America dominates the global market, thanks to:

• Robust healthcare infrastructure
• High disease awareness
• Strong pipeline of clinical trials
• Presence of leading pharmaceutical companies

For example:

• In November 2024, Attralus, Inc. received FDA orphan drug designation for its compound AT-02 for transthyretin-associated amyloidosis (ATTR).
• Around the same time, Ionis Pharmaceuticals and AstraZeneca received FDA approval for WAINUA (eplontersen), the first self-injectable therapy for hereditary ATTR with polyneuropathy (hATTR-PN).

Such regulatory milestones are helping the region maintain its leadership in rare disease therapeutics.

Key Players in the Amyloidosis Therapeutics Market

The market is competitive yet collaborative, with companies pursuing targeted drug discovery, precision therapies, and patient-centric care. Major players include:

• Pfizer Inc.
• Johnson & Johnson Services, Inc.
• Takeda Pharmaceutical Company Limited
• Amgen Inc.
• Alnylam Pharmaceuticals, Inc.
• AstraZeneca
• BridgeBio Inc.
• Ionis Pharmaceuticals
• Attralus, Inc.
• Eisai Co., Ltd.

These companies are partnering with research institutes, patient advocacy groups, and healthcare providers to expand access, improve outcomes, and accelerate innovation.

Challenges and the Road Ahead

While the future looks promising, the path forward isn’t without hurdles:

• High treatment costs can limit accessibility.
• Complexity of diagnosis often leads to delayed or missed treatment windows.
• The rarity of the disease makes large-scale clinical trials difficult.

However, as stakeholders align to address these challenges, the next decade is expected to bring about:

• More orphan drug approvals
• Increased reimbursement support
• Next-generation therapies using AI, genomics, and CRISPR

Conclusion: A Market Transforming Rare Disease Care

As awareness and diagnostics improve, and research accelerates, the amyloidosis therapeutics market is shifting from niche to notable. With promising drugs in the pipeline and expanding global interest, the industry is well-positioned to transform the lives of thousands affected by this complex condition.

Companies investing in this space are not just exploring a new revenue stream—they are shaping the future of rare disease care.

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