Amyloidosis Market Trends: How New Drug Approvals Are Changing Outcomes

Amyloidosis, a rare and often fatal disease characterized by the accumulation of misfolded proteins in organs and tissues, has long posed significant challenges in diagnosis and treatment. However, recent advancements in therapeutics have ushered in a new era, offering hope to patients and reshaping the market landscape. This blog delves into the latest trends in the amyloidosis market, focusing on how new drug approvals are changing outcomes for patients worldwide.

Understanding Amyloidosis

Amyloidosis encompasses a group of diseases where abnormal protein deposits, known as amyloids, accumulate in organs, impairing their function. The most common types include:

• AL (Light-chain) Amyloidosis: Caused by an abnormality in plasma cells producing light chains that form amyloid deposits.

• ATTR (Transthyretin) Amyloidosis: Results from misfolded transthyretin proteins, either due to genetic mutations (hereditary) or age-related changes (wild-type).

• AA (Secondary) Amyloidosis: Associated with chronic inflammatory conditions leading to serum amyloid A protein deposits.
  

Market Growth and Dynamics

The global amyloidosis treatment market has witnessed substantial growth, driven by increased awareness, improved diagnostic techniques, and the introduction of novel therapies. According to Precedence Research, the market was valued at USD 2.94 billion in 2024 and is projected to reach USD 6.45 billion by 2034, growing at a CAGR of 8.18% from 2025 to 2034.

Recent Drug Approvals Revolutionizing Treatment

1. Acoramidis (Attruby)

   In November 2024, the FDA approved Acoramidis (marketed as Attruby) for treating transthyretin amyloid cardiomyopathy (ATTR-CM). Developed by BridgeBio Pharma, Acoramidis is a near-complete transthyretin stabilizer that significantly reduces cardiovascular death and hospitalization rates. Clinical trials demonstrated its efficacy in improving patient outcomes, marking it as a significant advancement in ATTR-CM treatment.
   

2. Vutrisiran (Amvuttra)

   Alnylam Pharmaceuticals' Vutrisiran, approved for hereditary ATTR amyloidosis, has shown promise in treating ATTR-CM. A recent study revealed that Vutrisiran reduced deaths and cardiovascular events by 28% across all trial patients. Its quarterly dosing schedule offers a convenient option for patients, potentially improving adherence and quality of life.
   

3. Daratumumab (Darzalex Faspro)

   For AL amyloidosis, the FDA granted accelerated approval to Daratumumab in combination with other agents. This monoclonal antibody targets CD38 on plasma cells, reducing the production of amyloidogenic light chains. Clinical trials have shown rapid and deep hematologic responses, translating into improved organ function and survival rates.
   

Impact on Patient Outcomes

The introduction of these therapies has significantly improved patient outcomes:

• Enhanced Survival Rates: New treatments have led to a marked increase in overall survival, particularly in patients with cardiac involvement.

• Improved Quality of Life: Reduced hospitalization rates and symptom burden have enhanced daily functioning and well-being.

• Expanded Treatment Options: Patients now have access to multiple therapies, allowing for personalized treatment plans based on disease subtype and severity.
  

Challenges and Considerations

Despite these advancements, challenges remain:

• Cost and Accessibility: High treatment costs can limit access, especially in low- and middle-income countries. For instance, Tafamidis, another ATTR-CM treatment, carries an annual cost above $200,000 per patient.

• Diagnosis Delays: Early symptoms are often nonspecific, leading to delayed diagnosis and treatment initiation.

• Need for Further Research: Ongoing studies are essential to understand long-term effects, optimal treatment combinations, and management strategies for different amyloidosis types.
  

Future Outlook

The amyloidosis treatment landscape is poised for continued evolution:

• Emerging Therapies: Research into gene-silencing approaches and novel monoclonal antibodies holds promise for more effective treatments.

• Improved Diagnostics: Advances in imaging and biomarker identification will facilitate earlier diagnosis and monitoring.

• Global Market Expansion: As awareness grows and new treatments become available, the market is expected to expand, offering opportunities for pharmaceutical companies and improved care for patients worldwide.
  

Conclusion

The recent approvals of Acoramidis, Vutrisiran, and Daratumumab represent significant milestones in amyloidosis treatment, offering new hope to patients and transforming the market landscape. While challenges persist, continued innovation and collaboration among stakeholders promise a brighter future for those affected by this complex group of diseases. Please write to enquire@grgonline.com to learn how GRG Health is helping clients gather more in-depth market-level information on such topics.