Transthyretin Amyloidosis Treatment Market By Product Type (ATTR-CM, ATTR-PN), By Therapy (Targeted Therapy, Supportive Therapy), By Disease Type (Hereditary Amyloidosis, Wild-type Amyloidosis, Other), By Distribution Channel (Hospitals Pharmacy, Retail Pharmacies, Other), Global Market Size, Segmental analysis, Regional Overview, Company share analysis, Leading Company Profiles And Market Forecast, 2025 – 2035

Industry Outlook

The Transthyretin Amyloidosis Treatment Market accounted for USD 6.78 Billion in 2024 and is expected to reach USD 29.04 Billion by 2035, growing at a CAGR of around 14.14% between 2025 and 2035. The Transthyretin Amyloidosis (ATTR) Treatment Market focuses on therapies for ATTR amyloidosis, a rare and progressive disease caused by the misfolding of transthyretin (TTR) protein, leading to amyloid deposits in organs and tissues, affecting the heart, nerves, and other organs.

The condition is classified into hereditary (hATTR) and wild-type (wtATTR) amyloidosis, with treatment options including TTR stabilizers (tafamidis), gene silencing therapies (patisiran, vutrisiran), and TTR protein degradation approaches. The market is driven by rising prevalence, advancements in RNA interference (RNAi) therapies, increasing awareness, and growing R&D efforts. Additionally, ongoing clinical trials, regulatory approvals, and pharmaceutical collaborations are expanding treatment options. Key players in the market include Pfizer, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, BridgeBio Pharma, and Eidos Therapeutics.

Report Scope:

Market Dynamics

Rising Global Demand for Transthyretin Amyloidosis Treatment Due to Increasing Health Awareness and Advancements in Therapeutics

The global marketplace for transthyretin amyloidosis (ATTR) treatments expands because more patients adopt newly developed treatment approaches. New focused therapies continue to emerge because healthcare costs keep rising, and the population appreciates increasing awareness alongside biotech advancements. Throughout the medical field, patients, together with healthcare professionals, choose gene therapies RNA-targeted medications, and disease-modifying medicines rather than traditional options. Early intervention acceptance together with personalized medicine advances because countries experience population aging and advances in diagnostic methods. Medicinal innovations serve to raise survival rates prevent disease progression and simultaneously enhance patient health quality. Healthcare research advances as well as regulatory authorizations enhance access to markets through newly available treatments. The speed of innovation rises through the collaboration framework between pharmaceutical corporations and academic institutions. ATTR therapy receives additional innovative treatment options because clinical studies continue to grow. Healthcare statutes together with payment system operations determine how affordable treatment options become as well as their adoption rates.

Global strategic partnerships between companies extend patient reach into new markets and enhance market penetration. The market continues to grow over time because health expenditure for rare diseases continues to increase. Online technologies, together with social media, allow doctors to boost their engagement with patients and create more awareness. Research available in the market shows that specialized creative treatments for ATTR are gaining prominence. The expansion in market share falls under constant competition between new biotech startups and established pharmaceutical companies.

Growing Awareness of Transthyretin Amyloidosis Among Younger and At-Risk Populations

Patients, along with their caregivers, now pursue active searches for both rapid diagnosis and advanced treatment options that had not been common during previous times. Obtaining genetic tests for early illness detection becomes more accessible, thus leading to better patient outcomes through effective disease management. Patients gain important ATTR knowledge through digital health platforms as well as patient advocacy organizations and social media platforms. Younger patient populations along with caregivers advocate the development of new treatments because they value personal techniques that combine effectiveness and creativity. Scientific evidence indicates that the ATTR market continues to grow due to the rising trend of proactive healthcare and lifespan compatibility. Pharmaceutical organizations focus their investments on precision medicine and targeted medicines while working to enhance patient support systems because healthcare requirements change.

Regulatory agencies and healthcare providers also play a crucial role in expanding awareness by supporting educational initiatives and early screening programs. Increased funding for rare disease research has accelerated the development of novel therapies, making treatment options more effective and accessible. Telemedicine and digital consultations have further improved patient engagement, allowing individuals to seek expert opinions and disease management strategies remotely. Clinical trial participation has increased as more patients and caregivers become informed about emerging therapies through online platforms and advocacy groups. Collaborations between biotech firms and academic institutions continue to drive innovation in ATTR treatment, resulting in more personalized and targeted approaches. Growing media coverage and awareness campaigns have helped reduce misdiagnoses and delayed treatment, leading to earlier interventions and improved quality of life. As a result, pharmaceutical companies are expanding their global presence to cater to rising demand in underserved markets.

Advancements in Precision Medicine and Gene Therapy Offer Opportunities for Market Growth

Technical progress in precision medicine and gene therapy enables numerous chances to design improved and effective ATTR therapy approaches. Medical professionals work to discover superior therapeutic options than symptom management; thus, the market demands RNA-based treatments and both gene-silencing techniques and disease-altering drugs. The development of innovative treatments occurs because advanced biopharmaceutical research enables the reduction of disease progression while improving patient well-being.

New diagnostic techniques have the dual purpose of improving early and accurate ATTR identification so doctors can begin prompt intervention and create personalized treatment plans. The health-driven innovations in the ATTR sector provide benefits for people with diagnosed ATTR conditions and also assist healthcare professionals in testing vulnerable populations. The market expansion and worldwide disease management improvements stem from advancements that boost ATTR medicine efficiency together with patient accessibility and friendly treatment design.

Growing Patient Awareness and Improved Diagnostics Fuel Transthyretin Amyloidosis Treatment Market Growth

The market continuously grows because patients, combined with medical staff, are becoming more informed about ATTR transthyretin amyloidosis. The medical community achieves improved diagnosis rates and speedier reaction time because of population-wide early symptom recognition. Disease awareness has expanded through advocacy group educational efforts combined with medical research social media and public engagement activities.

Patients are now able to detect hereditary and wild-type ATTR at an earlier stage because genetic tests have become more accessible alongside improved imaging techniques. Soaring awareness about effective treatment options drives pharmaceutical companies to develop new remedies because the market demand continues to grow.

Emerging RNA-Targeted and Gene Therapies Revolutionize ATTR Treatment

The treatment options for ATTR disease now progress through the development of gene and RNA-targeted therapeutic methods. Research-based methods through antisense oligonucleotides combined with small interfering RNA demonstrate strong potential to reduce the production of aberrant transthyretin protein. South American patients with inherited ATTR now have access to gene-editing technology, which serves as a sustainable long-term treatment option.

Pharmaceutical companies dedicate substantial research investments to boost accessibility together with the safety and effectiveness of these therapies. The growing market stability results from new scientific discoveries that lead to more advanced personalized therapies being cleared by regulatory bodies for ATTR patients.

Industry Experts Opinion

“The Transthyretin Amyloidosis Treatment Market is evolving beyond symptom management toward innovative, targeted therapies. Advancements in gene and RNA-based treatments are transforming patient care, offering new hope for improved outcomes and quality of life."

• Dr. Michael Reynolds, CEO, BioMed Therapeutics

"The future of Transthyretin Amyloidosis treatment lies in precision medicine and breakthrough therapies. With growing awareness and innovation, we are redefining disease management and patient outcomes."

• Dr. Emily Carter, Chief Medical Officer, NeuroGen Pharma

Segment Analysis

Based on the Product Type, the market for treatments for transthyretin amyloidosis (ATTR) is divided into two segments: ATTR-CM (transthyretin amyloid cardiomyopathy) and ATTR-PN (transthyretin amyloid polyneuropathy). ATTR-CM develops because amyloid induces wall stiffness in the heart, which leads to heart failure. The classification system features hATTR-CM as genetic and wtATTR-CM as the age-related form. The symptoms of this condition include both leg edema and exhaustion as well as arrhythmias and shortness of breath. ATTR-PN primarily affects neurological functions by causing autonomic dysfunction along with gastrointestinal problems and muscular weakness followed by numbness and developing nerve deterioration. Hereditary ATTR commonly appears together with the disease (hATTR-PN). Different treatments are used for each illness type, so cardioprotective drugs treat ATTR-CM, but neuropathy-targeted medications are necessary for treating ATTR-PN.

Based on the Distribution Channel, The market for transthyretin amyloidosis (ATTR) treatments is divided into segments based on the distribution channels, guaranteeing that patients may obtain necessary drugs in a variety of ways. Most sophisticated RNA-targeted and gene-silencing treatments that handle ATTR conditions undergo distribution through hospital pharmacies under medical supervision. Patients experience better disease management through retail pharmacy access to symptomatic therapies alongside stabilizer drugs, which they can control easily. The rise of direct-to-patient delivery services along with specialty clinics and internet pharmacies has been driving more patients to use alternate distribution methods for expensive particular treatments. Shorter and simpler refills are available through online platforms to patients who need regular prescription refills, yet specialty pharmacies secure access to hard-to-find treatments for rare medical conditions. These separate distribution channels work together to offer essential healthcare access for patients.

Regional Analysis

Transthyretin amyloidosis (ATTR) treatment markets primarily operate in North America because the region has an advanced healthcare system with great awareness levels and high rates of early diagnosis. The market controls the market space because essential pharmaceutical industries exist along with substantial research investments and favored reimbursement rules. The USA position dominates the area by developing TTR stabilizers together with gene-silencing and RNA-targeted therapies. The increase of market size stems from clinical research together with FDA regulatory approval of new treatment options. Stronger treatment outcomes become possible when ATTR-focused facilities and well-trained medical staff are available to patients.

The ATTR Treatment Market in Asia-Pacific demonstrates the most rapid expansion because governments boost support for rare disease medical care, and patients obtain better healthcare access combined with improved disease awareness. The number of ATTR-diagnosed cases is increasing in China, India, Japan, and South Korea because of the development of genetic testing and imaging technologies. The pharmaceutical industry deepens its presence through clinical studies and strategic partnerships, and public institutions and healthcare systems jointly finance evaluations for early detection and investigations of rare diseases. Forecasted market growth in the region will be driven by two factors: the rapidly developing biopharmaceutical industry and the increasing healthcare costs. Japan and other nations in Asia-Pacific will become significant contributors to the global ATTR market due to expanding patient treatment choices of innovative medical therapies.

Competitive Landscape

The Transthyretin Amyloidosis (ATTR) Treatment Market experiences intense market competition as pharmaceutical companies attempt numerous innovations to acquire regulatory clearances for their therapies. The market for ATTR treatment is concentrated among Pfizer, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, and AstraZeneca because of their commitment to research their strong strategic alliances, and their development of advanced medicine solutions. The ATTR leadership company Pfizer uses two TTR stabilizer compounds named Tindamax and Vyndaqel (tafamidis) in their product line. The company directs its efforts toward clinical trial development and programs that raise patient awareness to expand its market reach.

RNA-based therapy specialist Alnylam Pharmaceuticals has achieved major market success with its therapeutic candidates Onpattro (patisiran) and Amvuttra (vutrisiran), which suppress the TTR gene to minimize amyloid accumulation in patient cells.Under a partnership with AstraZeneca, Ionis Pharmaceuticals develops Eplontersen, which represents an investigational RNA-targeted medication for ATTR treatment. Research and development of this treatment aims to create more effective care for patients suffering from ATTR. Eligibility approval mechanisms along with trial-stage advances guide market evolution as companies work through strict official hurdles to create their competitive advantages. The ATTR Treatment Market exists under a combination of continuous innovation and strategic partnerships and also encounters regulatory hurdles. Companies continue their focus on creating advanced gene-silencing treatments while developing improved access to therapy and stronger patient results to increase market competition.

Transthyretin Amyloidosis Treatment Market, Company Shares Analysis, 2024

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Recent Developments:

• In October 2024, AstraZeneca finalized the acquisition of Ionis Pharmaceuticals' Eplontersen program for $1.2 billion, reinforcing its commitment to advancing RNA-based therapeutics for ATTR treatment. This acquisition aligns with AstraZeneca’s strategy to develop next-generation gene-silencing therapies with improved efficacy and patient outcomes, further intensifying competition in the ATTR market.
• In March 2024, Alnylam Pharmaceuticals announced expanded regulatory approvals for Amvuttra (vutrisiran) in Europe, the Middle East, and Africa (EMEA), enhancing patient access to RNA-silencing therapy for ATTR-PN. This expansion aims to strengthen Alnylam’s presence in global markets and address the growing demand for innovative treatment options.

Report Coverage:

By Product Type

• ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
• ATTR-PN (Transthyretin Amyloid Polyneuropathy)

By Therapy

•  Targeted Therapy
•  Supportive Therapy

By Disease Type

• Hereditary Amyloidosis
• Wild-type Amyloidosis
• Other

By Distribution Channel

• Hospitals Pharmacy
• Retail Pharmacies
• Other

By Region

North America

• U.S
• Canada

Europe

• U.K.
• France
• Germany
• Spain
• Rest of Europe
• Italy

Asia Pacific

• China
• Japan
• India
• Australia
• South Korea
• Singapore
• Rest of Asia Pacific

Latin America

• Brazil
• Argentina
• Mexico
• Rest of Latin America

Middle East & Africa

• GCC Countries
• South Africa
• Rest of Middle East & Africa

List of Companies:

• Alnylam Pharmaceuticals
• Bridgebio Pharma
• Pfizer
• Ionis Pharmaceuticals
• Astrazeneca
• Alexion Pharmaceutical
• Proliferan
• Enobio Pharma
• Moderna
• Intellia Therapeutics
• Regeneron Pharmaceuticals
• SomaLogic
• Prothena Corporation
• Eidos Therapeutics
• Corina Therapeutics

Frequently Asked Questions (FAQs)