Transthyretin amyloid cardiomyopathy, commonly known as ATTR-CM, is a rare progressive heart disease caused by the buildup of abnormal deposits of a protein called transthyretin in the heart. The deposits interfere with the normal working of the heart and worsen heart failure over time. Currently, patisiran (Onpattro) and tafamidis (Vyndaqel, Vyndamax) are approved for the treatment of ATTR-CM. However, several biotech companies are exploring newer treatment approaches such as gene therapy and gene silencing through RNA interference technology, which have shown promising results in clinical trials.
The global transthyretin amyloid cardiomyopathy treatment market is estimated to be valued at US$ 1943.69 billion in 2024 and is expected to exhibit a CAGR of 8.0% over the forecast period 2024 to 2031.
Key Takeaways
Key players operating in the transthyretin amyloid cardiomyopathy treatment market are Pfizer Inc., Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Eidos Therapeutics, and Prothena.
The market is witnessing significant growth due to the rising prevalence of ATTR-CM worldwide. As per estimates, over 400,000 people are afflicted by ATTR amyloidosis globally. Furthermore, the disease often remains undiagnosed resulting in an increase in number of cases.
Novel treatment approaches such as gene therapy and gene silencing are being explored actively which can potentially provide a long-term cure and drive the Transthyretin Amyloid Cardiomyopathy Treatment Market Growth . For instance, Pfizer is developing a gene therapy approach PF-07258669 which has shown encouraging early results.
Market Trends
Growing preference for advanced therapeutics - Development of advanced therapeutics such as gene therapies or gene silencing technologies that target the root cause of ATTR-CM is expected to revolutionize the management of the disease. Several biopharma companies are developing novel therapies to replace lifelong administration of medicines.
Increasing focus on early diagnosis - Late diagnosis often results in worsening of symptoms and reduced treatment effectiveness. R&D activities in the field of diagnostic markers and non-invasive tests are being ramped up to enable early detection of ATTR-CM. This will facilitate timely intervention and improve clinical outcomes.
Market Opportunities
Large patient pool in undiagnosed/underdiagnosed regions - Due to lack of awareness and diagnostic challenges, ATTR-CM remains highly underdiagnosed, especially in developing countries. Creating screening programs and diagnostic infrastructure can help tap into this sizeable hidden patient population.
Combination therapy approaches - Exploring synergistic combinations of approved drugs or drugs with different mechanisms can enhance treatment efficacy. Combining small molecule drugs with gene therapies is a promising avenue.
Impact of COVID-19 on Transthyretin Amyloid Cardiomyopathy Treatment Market Growth
The outbreak of COVID-19 pandemic has significantly impacted the growth of transthyretin amyloid cardiomyopathy treatment market globally. The imposition of nationwide lockdowns and movement restrictions disrupted the supply chain and delayed production schedules. The supply constraints hampered the availability of diagnostics tests and treatment drugs required for effective management of ATTR-CM. The hospitals allocated maximum resources towards treatment of COVID infected patients which reduced the focus on ATTR-CM management. The fear of infection transmission compelled many patients to defer or cancel their routine checkups and scheduled therapies. This negatively impacted the new patient registrations and diagnostic procedures during 2020 and 2021.
The restrictions on international travel and transport also disrupted the clinical trials of novel treatment therapies in late-stage development pipeline. Many pharma companies faced challenges in patient recruitment and data collection efforts. However, with rapid vaccination drives and relaxed containment measures in 2022, the market is recovering fast. The focus on ATTR-CM is now regaining as medical resources are available. The diagnostic procedures and treatment rates have bounced back to pre-pandemic levels. The pharma vendors are accelerating their product development plans and clinical trials are back on track. With the learnings from the pandemic, healthcare systems are better prepared with remote monitoring technologies to ensure continuity of care. The long-term demand outlook remains positive, though short-term growth was affected in 2020-21 period due to COVID-19 disruptions.
Geographical Concentration of Transthyretin Amyloid Cardiomyopathy Treatment Market in Terms of Value
In terms of value, the North America region has historically been the largest market for transthyretin amyloid cardiomyopathy treatment globally. High disease prevalence combined with high per capita healthcare spending in the United States has ensured significant market value concentration in North America. As per estimates, more than 50% of the global ATTR-CM treatment market value in 2024 can be attributed to the North America region led by the US. Increased awareness, early diagnosis and widespread insurance coverage for expensive novel drugs are supporting strong demand in the US. Europe is the second largest geographical market given developed healthcare infrastructure and reimbursement programs across major countries. However, the Asia Pacific region is emerging as the fastest growing market given rising investments to strengthen healthcare systems across developing nations. Rising income levels are also driving higher patient affordability for advanced therapies in Asia Pacific. This makes it an important region to focus for long term market opportunities in ATTR-CM treatment space.
Fastest Growing Region for Transthyretin Amyloid Cardiomyopathy Treatment Market
The Asia Pacific region is projected to be the fastest growing geographical market for transthyretin amyloid cardiomyopathy treatment globally through 2031. The lucrative growth opportunities can be attributed to growing disease prevalence aided by aging populations, economic development and lifestyle changes in countries like China and India. The growing healthcare spending by governments to strengthen public insurance programs will enhance access to advanced diagnostics and therapies. Rising medical tourism and presence of large patient pools will attract global pharma investments into local drug production and clinical trials. This will help address the current unmet need and reduce dependence on imported drugs. Increasing collaborations between multinational hospitals and local centers to spread awareness and conduct industry-sponsored awareness campaigns will further augment early detection rates. In addition, the low market maturity offers huge potential for penetration of innovative therapeutic options. All these factors will drive strong double digit growth rates for the Asia Pacific ATTR-CM treatment market over the next decade.
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Money Singh is a seasoned content writer with over four years of experience in the market research sector. Her expertise spans various industries, including food and beverages, biotechnology, chemical and materials, defense and aerospace, consumer goods, etc. (https://www.linkedin.com/in/money-singh-590844163)
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