Report Overview

According to the deep-dive market assessment study by Growth Plus Reports, the global transthyretin amyloidosis (ATTR) therapeutics market was valued at US$ 4.5 billion in 2021 and is expected to register a revenue CAGR of 7.8% to reach US$ 9.53 billion by 2030.

Transthyretin Amyloidosis (ATTR) Therapeutics Market Fundamentals

Transthyretin amyloidosis is a progressive condition that comprises abnormal deposition of aberrant proteins under the skin or in organs, such as kidneys, heart, eyes, brain, or tissue organs affecting the peripheral nervous system. Transthyretin amyloid polyneuropathy and transthyretin amyloid cardiomyopathy are the two forms of illnesses. It is a rare metabolic disorder that compromises organ and tissue function and structure, resulting in potentially fatal illnesses. Amyloidosis is a disorder that arises when amyloid protein builds up in tissues and organs. Furthermore, the symptoms can worsen with time and vary depending on the protein deposition. Amyloid buildup in the nerves can permanently damage the neurological system and eventually cause loss of sensation in the limbs, hands, and feet.

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Transthyretin Amyloidosis (ATTR) Therapeutics Market Dynamics 

Demand for transthyretin amyloidosis drugs has increased in the recent few years owing to its rising usage in disease management. This factor is driving the global transthyretin amyloidosis therapeutics market revenue growth. Moreover, growing awareness regarding the disease is expected to further support the transthyretin amyloidosis therapeutics market revenue growth.

Due to the rarity of the condition, currently, there are limited options for transthyretin amyloidosis treatment. This factor, in turn, is estimated to drive the transthyretin amyloidosis therapeutics market revenue growth. Furthermore, improved diagnostic methods, rapidly developing healthcare infrastructure, and increasing healthcare expenditures facilitate market revenue growth to some extent.

An increase in R&D initiatives aimed at developing novel diagnostic tools and multimodal therapy approaches is expected to result in a higher survival rate and a better prognosis for transthyretin amyloidosis. For instance, in November 2019, the Amyloidosis Research Consortium created a disease-specific patient-reported outcome (PRO) instrument to understand and assess the impact of transthyretin amyloidosis from the patient’s perspective. An introduction of such a tool is expected to improve the research in producing a new target-specific medicine by understanding disease complexity and patients’ need, consequently boosting the revenue growth opportunities in the transthyretin amyloidosis therapy market. Additionally, strategic activities performed by major participants, such as spreading awareness about target disorders, such as ATTR, and the long-term advantages of existing medicines, are likely to fuel market revenue expansion in the next few years.

As per the National Organization for Rare Disorders (NORD), transthyretin amyloidosis is more common in African Americans, and the prevalence is 1 in 100,000 individuals among Caucasians. The high cost of liver transplantation is a key restraint to the transthyretin amyloidosis therapeutics market revenue.

Transthyretin Amyloidosis (ATTR) Therapeutics Market Ecosystem

The global transthyretin amyloidosis therapeutics market has been analyzed from four perspectives: disease type, drug class, distribution channel, and region.

Transthyretin Amyloidosis Therapeutics Market by Disease Type

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Based on the disease type, the global transthyretin amyloidosis therapeutics market is segmented into familial type and wild type.

The familial (hereditary) transthyretin amyloidosis segment accounts for the largest revenue share of the global market. Familial (hereditary) transthyretin-mediated amyloidosis is an autosomal dominant, deadly illness that affects several organs and causes death and disability. Pharmaceutical companies are constantly working on drug research to treat this disorder, and several treatments are still in the pipeline for commercialization.

Transthyretin Amyloidosis Therapeutics Market by Drug Class

Based on the drug class, the global transthyretin amyloidosis therapeutics market is segmented into siRNA agents, antisense oligonucleotides, and transthyretin stabilizers. 

The siRNA agents segment is expected to demonstrate lucrative revenue growth during the forecast period. This can be attributed to highly effective results of siRNA agents and increasing R&D activities related to these agents. 

Transthyretin Amyloidosis Therapeutics Market by Distribution Channel 

Based on the distribution channel, the global transthyretin amyloidosis therapeutics market is segmented into hospital, retail, and online pharmacies. 

The hospital segment is expected to dominate the global market during the forecast period. The majority of hospitals are engaged in disease diagnosis, research, and treatment. For instance, Brigham and Women's Hospital in Boston, Massachusetts, concentrates on misdiagnosed disorders. It has established the Amyloidosis Program, which offers access to innovative medicines, patient-centered care, and new clinical trials. The experts educate physicians and patients and increase awareness among patients. These activities are expected to accelerate the segment’s revenue growth in the next few years.

Transthyretin Amyloidosis Therapeutics Market by Region 

Based on the region, the global transthyretin amyloidosis therapeutics market is segmented into North America, Europe, Asia Pacific, and the Rest of the World. 

North America dominates the global market due to increasing novel pharmaceuticals prescription, attractive reimbursement policies, and higher treatment rates in the region. The introduction of novel medicines in the region is a key market revenue driver.

Transthyretin Amyloidosis (ATTR) Therapeutics Market Competitive Landscape 

The prominent players in the global transthyretin amyloidosis therapeutics market include: 

• Pfizer Inc.
• Alnylam Pharmaceuticals, Inc.
• Ionis Pharmaceuticals 
• Prothena
• SOM BIOTECH
• AstraZeneca
• Eidos Therapeutics, Inc
• Novo Nordisk A/S

Transthyretin Amyloidosis (ATTR) Therapeutics Market Strategic Developments 

• In June 2022, the U.S. FDA approved AMVUTTRATM (vutrisiran), which is an RNAi therapeutic administered via subcutaneous injection once every 3 months for the treatment of polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis in adults, according to Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company. hATTR amyloidosis is a rare, hereditary, fast-developing, and lethal disease marked by catastrophic polyneuropathy symptoms and few therapy possibilities.
• In December 2021, Pfizer Inc. announced a post-hoc interim study demonstrating that treatment with VYNDAQEL (tafamidis meglumine)/VYNDAMAX (tafamidis) showed a clinically significant survival advantage for individuals with transthyretin amyloid cardiomyopathy after 5 years (ATTR-CM). The ATTR-ACT Phase 3 Transthyretin Amyloid Cardiomyopathy Clinical Trial (ATTR-ACT) and its Long-Term Extension (LTE) research were examined in this study.
• In December 2021, AstraZeneca and Ionis Pharmaceuticals, Inc. (Ionis) inked a new global development and commercialization agreement for eplontersen, formerly known as IONIS-TTR-LRX. Eplontersen is a ligand-conjugated antisense medication that was in Phase III clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-P) (ATTR-PN). It treats both hereditary and non-hereditary forms of TTR amyloidosis by lowering the production of transthyretin (TTR protein) (ATTR).
• In May 2021, Janssen Pharmaceutical Companies of Johnson & Johnson released updated results from the Phase 3 ANDROMEDA study, which evaluated DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) for the treatment of patients with newly diagnosed light chain (AL) amyloidosis, a rare blood cell disorder associated with organ deterioration, most notably the heart, kidneys, and liver. Longer-term results from a median follow-up of 20.3 months revealed that patients who received DARZALEX FASPRO in combination with bortezomib (VELCADE), cyclophosphamide, and dexamethasone (D-VCd) had significantly higher occurrences of hemCR than those who received VCd alone.

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