Global Transthyretin Amyloidosis Therapeutics Market Growth, Share, Size, Trends and Forecast (2025 - 2031)

• In June 2022, Alnylam announced FDA approval of AMVUTTRA (Vutrisiran), an RNAi therapeutics for the treatment of the Polyneuropathy of Hereditary Transthyretin-mediated Amyloidosis in adults. AMUVUTTRA (Vutrisiran) is the first and only FDA-approved drug demonstrating reversal in neuropathy impairment with SC administration once every three months.

• In January 2022, AstraZeneca announced Orphan Drug Designation for Eplontersen in the US. Eplontersen is an investigational antisense medicine designed to treat transthyretin amyloidosis (ATTR), a systemic, progressive, and fatal disease. The medicine helps to reduce the production of transthyretin (TTR protein) for the treatment of both hereditary and non-hereditary forms of ATTR.

The global transthyretin amyloidosis therapeutics market is experiencing a significant upsurge, primarily driven by advancements in medical research and increasing awareness among healthcare professionals and patients alike. Transthyretin amyloidosis, a rare and potentially fatal disease characterized by the deposition of abnormal amyloid protein in various tissues and organs, presents a pressing need for effective treatment options. As such, pharmaceutical companies are intensifying their efforts to develop innovative therapies targeting this debilitating condition.

One key factor propelling market growth is the expanding pipeline of novel drugs specifically designed to address transthyretin amyloidosis. These therapies aim to either stabilize transthyretin protein or inhibit its production, thereby slowing down disease progression and improving patient outcomes. Moreover, the approval and commercialization of new drugs have bolstered the market landscape, offering patients additional treatment choices and fostering competition among pharmaceutical manufacturers.

Another driving force behind the market's expansion is the growing prevalence of transthyretin amyloidosis worldwide, particularly among aging populations. As life expectancy increases and diagnostic techniques become more sophisticated, the incidence of this rare disease is expected to rise, creating a substantial market opportunity for therapeutics. Additionally, the implementation of supportive policies and initiatives by regulatory authorities and healthcare organizations is further fueling market growth by streamlining drug development processes and facilitating market access for novel treatments.

Despite these promising trends, the transthyretin amyloidosis therapeutics market faces several challenges, including high treatment costs, limited patient access to specialized care, and the need for improved diagnostic tools. Addressing these hurdles will be crucial for sustaining market growth and ensuring that patients receive timely and effective interventions. Overall, with ongoing research efforts, technological advancements, and collaborative initiatives across the healthcare sector, the global transthyretin amyloidosis therapeutics market is poised for continued expansion in the foreseeable future.

Global Transthyretin Amyloidosis Therapeutics Segment Analysis

In this report, the Global Transthyretin Amyloidosis Therapeutics Market has been segmented by Drugs, Disease Type, Distribution Channel and Geography.

Global Transthyretin Amyloidosis Therapeutics Market, Segmentation by Drugs

The Global Transthyretin Amyloidosis Therapeutics Market has been segmented by Drugs into Tafamidis (Vyndaqel), Patisiran (ONPATTRO), Inotersen (TEGSEDI) and Others.

Tafamidis, marketed under the brand name Vyndaqel, is one of the leading drugs in this segment. It is a medication that stabilizes transthyretin, thereby slowing the progression of the disease. Tafamidis has shown promising results in clinical trials and has gained approval in several regions, contributing substantially to the overall revenue of the transthyretin amyloidosis therapeutics market.

Another significant drug in this segment is Patisiran, marketed as ONPATTRO. Patisiran is an RNA interference (RNAi) therapeutic designed to target and inhibit the production of abnormal transthyretin protein, which is a hallmark of transthyretin amyloidosis. With its unique mechanism of action, Patisiran offers a novel approach to treating this condition and has demonstrated efficacy in clinical studies, leading to its approval and widespread use.

Inotersen, commercially known as TEGSEDI, is also a notable drug used in the management of transthyretin amyloidosis. It works by inhibiting the production of transthyretin protein in the liver, thereby reducing the accumulation of amyloid deposits in various tissues and organs. Inotersen has shown efficacy in improving neurological symptoms and quality of life in patients with this debilitating disease.

The market segment includes other drugs that are either in the pipeline or approved for the treatment of transthyretin amyloidosis. These may include investigational therapies targeting different aspects of the disease pathology or alternative treatment options for patients who may not respond adequately to existing medications. Overall, the segmentation by drugs underscores the diversity and innovation in the development of therapeutics for transthyretin amyloidosis, offering hope for improved outcomes and better quality of life for affected individuals.

Global Transthyretin Amyloidosis Therapeutics Market, Segmentation by Disease Type

The Global Transthyretin Amyloidosis Therapeutics Market has been segmented by Disease Type into Hereditary Transthyretin Amyloidosis and Wild Type Amyloidosis.

Hereditary transthyretin amyloidosis (hATTR), also known as familial amyloid polyneuropathy (FAP), is caused by genetic mutations that lead to the production of abnormal transthyretin (TTR) protein. These mutations are inherited in an autosomal dominant manner, meaning that a single copy of the mutated gene from either parent is sufficient to cause the disease. Patients with hATTR typically experience progressive neurological symptoms, such as peripheral neuropathy, autonomic dysfunction, and cardiomyopathy. Given the genetic basis of hATTR, targeted therapies aimed at reducing the production or stabilizing the TTR protein have emerged as promising treatment options.

Wild type amyloidosis (ATTRwt) occurs in individuals without a known genetic predisposition and is primarily associated with aging. While the exact mechanisms underlying ATTRwt remain elusive, it is believed that age-related changes in the structure and function of TTR may contribute to the formation of amyloid deposits. Unlike hATTR, which predominantly affects the peripheral nervous system, ATTRwt often involves the heart, leading to a condition known as senile systemic amyloidosis (SSA). Management strategies for ATTRwt focus on symptom relief, organ-specific interventions, and supportive care measures to improve patients' quality of life and slow disease progression.

Global Transthyretin Amyloidosis Therapeutics Market, Segmentation by Distribution Channel

The Global Transthyretin Amyloidosis Therapeutics Market has been segmented by Distribution Channel into Hospital Pharmacies, Specialty Pharmacies, Retail Pharmacies and Online Pharmacies.

Hospital pharmacies play a crucial role in distributing transthyretin amyloidosis therapeutics, as hospitals are often the primary point of care for patients suffering from serious medical conditions. These pharmacies are equipped to handle a wide range of medications, including those required for treating ATTR, and they provide easy access to these therapeutics for both inpatient and outpatient settings.

Specialty pharmacies cater specifically to patients with complex or chronic conditions, such as transthyretin amyloidosis. These pharmacies have specialized knowledge and expertise in managing such conditions, providing personalized care and support services to patients. They ensure the availability of specialized medications and often offer delivery services to enhance convenience for patients.

Retail pharmacies serve as accessible points of distribution for transthyretin amyloidosis therapeutics, reaching a broader patient population. Patients can easily access these pharmacies in their local communities, making it convenient for them to refill prescriptions and seek advice from pharmacists regarding their medication regimens.

The emergence of online pharmacies has revolutionized the distribution of healthcare products, including transthyretin amyloidosis therapeutics. Online pharmacies offer the convenience of ordering medications from the comfort of one's home, with doorstep delivery services expanding access to treatment options for patients living in remote areas or those with mobility issues.

Global Transthyretin Amyloidosis Therapeutics Market, Segmentation by Geography

In this report, the Global Transthyretin Amyloidosis Therapeutics Market has been segmented by Geography into five regions; North America, Europe, Asia Pacific, Middle East and Africa and Latin America.

Global Transthyretin Amyloidosis Therapeutics Market Share (%), by Geographical Region, 2024

North America holds a prominent position in the global transthyretin amyloidosis therapeutics market, owing to the presence of well-established healthcare infrastructure, high adoption rates of novel treatment options, and a large patient pool. Additionally, extensive research and development activities, coupled with favorable reimbursement policies, further contribute to the market growth in this region. The increasing prevalence of ATTR, particularly in the aging population, is also a significant factor driving market expansion in North America.

Europe represents another significant market for transthyretin amyloidosis therapeutics, characterized by the presence of advanced healthcare systems and increasing awareness among healthcare professionals and patients regarding the disease. Countries such as the United Kingdom, Germany, France, and Italy are witnessing a surge in the demand for innovative therapeutics for the management of ATTR, supported by initiatives aimed at improving disease awareness and diagnosis rates.

The Asia Pacific region is poised to exhibit substantial growth in the transthyretin amyloidosis therapeutics market, attributed to the rapidly evolving healthcare infrastructure, expanding patient population, and increasing healthcare expenditure. Emerging economies such as China, India, and Japan are witnessing a rising prevalence of ATTR, driving the demand for effective treatment options and creating opportunities for market players to expand their presence in the region.

The Middle East and Africa, along with Latin America, are also expected to contribute to the growth of the global transthyretin amyloidosis therapeutics market. Factors such as improving access to healthcare services, growing investments in healthcare infrastructure, and rising awareness about rare diseases among healthcare providers and patients are anticipated to fuel market expansion in these regions. However, challenges related to limited healthcare access and affordability may hinder market growth to some extent in certain parts of these regions.

This report provides an in depth analysis of various factors that impact the dynamics of Global Transthyretin Amyloidosis Therapeutics Market. These factors include; Market Drivers, Restraints and Opportunities Analysis.

Drivers

• Aging Population
• Advancements in Treatment Options
• Increasing Awareness and Diagnosis

• Rising Research and Development Investments: The global transthyretin amyloidosis therapeutics market is witnessing a significant surge in research and development investments, driven by a growing understanding of the disease's mechanisms and an increasing recognition of its prevalence. Transthyretin amyloidosis, a rare and progressive condition characterized by the accumulation of abnormal amyloid protein deposits, poses substantial challenges to patients and healthcare providers alike. However, recent advancements in molecular biology and drug development techniques have fueled optimism within the medical community regarding the potential for innovative treatment modalities.

  One key driver behind the escalating investments in transthyretin amyloidosis therapeutics is the pressing need for more effective treatment options. Current approaches primarily focus on symptom management and disease stabilization, leaving substantial room for improvement in terms of disease modification and patient outcomes. As researchers delve deeper into the underlying molecular pathways of transthyretin amyloidosis, novel therapeutic targets are being identified, paving the way for the development of innovative drugs that aim to halt or reverse disease progression.

  The growing prevalence of transthyretin amyloidosis, particularly in aging populations, underscores the urgency of addressing unmet medical needs in this space. With an increasing number of patients being diagnosed with the disease worldwide, there is a corresponding rise in demand for therapeutic interventions that can offer meaningful clinical benefits. Consequently, pharmaceutical companies, biotechnology firms, and academic research institutions are ramping up their investments in translational research, clinical trials, and drug discovery initiatives aimed at bringing new and improved treatments to market. Overall, the escalating research and development investments in the global transthyretin amyloidosis therapeutics market hold promise for transforming the landscape of disease management and improving the quality of life for patients affected by this debilitating condition.

Restraints

• Limited Availability of Approved Therapies
• High Treatment Costs
• Lack of Effective Disease Management

• Regulatory Challenges and Approvals: The global transthyretin amyloidosis therapeutics market has witnessed significant advancements in recent years, but regulatory challenges persist in bringing new treatments to market. Transthyretin amyloidosis is a rare disease characterized by the accumulation of abnormal deposits of the protein transthyretin in various tissues and organs. Developing effective therapies for this condition requires navigating a complex regulatory landscape, as companies must demonstrate both safety and efficacy to gain approval from regulatory agencies such as the FDA and EMA.

  One of the primary regulatory challenges in the transthyretin amyloidosis therapeutics market is the small patient population. Because the disease affects a relatively limited number of individuals, conducting clinical trials with sufficient statistical power can be difficult. This challenge is compounded by the heterogeneity of the disease, which can make it challenging to identify suitable endpoints for clinical trials. Regulatory agencies may require innovative trial designs and endpoints to address these challenges and ensure that new therapies meet the needs of patients.

  Despite these challenges, regulatory agencies have shown a willingness to collaborate with drug developers to expedite the approval of promising therapies for transthyretin amyloidosis. Breakthrough therapy designation, orphan drug status, and accelerated approval pathways are among the regulatory mechanisms available to facilitate the development and approval of treatments for rare diseases. Additionally, regulatory agencies may provide guidance and support to companies throughout the drug development process, helping to streamline the path to approval. As our understanding of transthyretin amyloidosis continues to evolve, regulatory agencies and drug developers must work together to overcome challenges and bring innovative therapies to patients in need.

Opportunities

• Emerging Markets Expansion
• Targeted Therapy Development
• Genetic Screening Advancements

• Collaborative Research Initiatives: Collaborative research initiatives are becoming increasingly vital in advancing the treatment landscape for global transthyretin amyloidosis (ATTR). With this debilitating disease impacting individuals worldwide, pooling resources and expertise through collaborative efforts can accelerate the development of novel therapeutics. One significant aspect of these initiatives involves bringing together pharmaceutical companies, academic institutions, and research organizations to share knowledge, resources, and data. By fostering collaboration, researchers can leverage diverse perspectives and methodologies to address the multifaceted challenges posed by ATTR.

  Collaborative research initiatives play a crucial role in enhancing clinical trial design and execution. Given the complexity of ATTR and the need for rigorous clinical evaluation of potential therapies, collaborative efforts can facilitate the recruitment of larger and more diverse patient populations across multiple geographical regions. This inclusivity not only improves the generalizability of trial results but also ensures that diverse patient demographics are adequately represented, ultimately leading to more robust and reliable outcomes.

  Collaborative research initiatives pave the way for the discovery of innovative therapeutic approaches, such as gene therapies, RNA interference, and small molecule inhibitors. By fostering interdisciplinary collaboration among experts in genetics, biochemistry, pharmacology, and other relevant fields, these initiatives enable the exploration of novel drug targets and mechanisms of action. Additionally, collaborative efforts can expedite the translation of preclinical discoveries into clinical applications by providing the necessary infrastructure and funding support. Overall, collaborative research initiatives are instrumental in driving progress in the development of effective therapies for global transthyretin amyloidosis, offering hope to patients and caregivers worldwide.

Key players in Global Transthyretin Amyloidosis Therapeutics Market include:

• Pfizer Inc
• Alnylam Pharmaceuticals, Inc
• Ionis Pharmaceuticals, Inc
• Akcea Therapeutics, Inc
• Eidos Therapeutics
• Prothena Corporation plc
• GlaxoSmithKline plc
• Ionis Pharmaceuticals
• Arcturus Therapeutics Holdings Inc
• FoldRx Pharmaceuticals

In this report, the profile of each market player provides following information:

• Company Overview and Product Portfolio
• Key Developments
• Financial Overview
• Strategies
• Company SWOT Analysis