Transthyretin Amyloidosis Treatment Market Size, Trends, Share, Growth | Report 2022-2030

The global transthyretin amyloidosis treatment market size was estimated at around USD 4.87 billion in 2021 and it is projected to hit around USD 9.35 billion by 2030, growing at a CAGR of 7.52% from 2022 to 2030.

Report Highlights

• ATTR-CM dominated the market for transthyretin amyloidosis and accounted for a revenue share of more than 80.11% in 2021. 
• Hereditary transthyretin amyloidosis captured the largest revenue share of over 60.21% in 2021. 
• Hospital pharmacies captured the largest revenue share of over 30.08% in 2021. 
• The targeted therapy segment dominated the market and accounted for a revenue share of over 90.17% in 2021. 
• North America dominated the market with a revenue share of over 65.02% in 2021 and the region is anticipated to maintain its dominance during the forecast period. 

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The transthyretin amyloidosis treatment market is mainly driven by the presence of supportive reimbursement programs and the growing African American population. The increasing African American population acted as a catalyst for the industry growth throughout the period owing to the high susceptibility to ATTR amyloidosis. The Val122Ile is the most prevalent mutation worldwide that leads to cardiomyopathy among the population specific to aged 65 years and above. Furthermore, this Val122Ile mutation is present in 3% to 4% of the African American population worldwide.

An increase in research activities for the development of novel diagnostic techniques and multidisciplinary therapeutic approaches is expected to lead to an increased survival rate and a more favorable prognosis of transthyretin amyloidosis. For instance, in November 2019, the Amyloidosis Research Consortium developed a disease-specific patient-reported outcome (PRO) tool to understand and measure the impact of transthyretin amyloidosis from the patient perspective. An introduction of such a tool will boost the research in developing a new target-specific drug by understanding disease complexity and patients’ need, thereby widening the opportunity in the transthyretin amyloidosis treatment market.

Strategic initiatives undertaken by major players such as creating awareness regarding the target conditions such as ATTR and the long-term benefits of available therapies are expected to fuel industry growth. For instance, in 2019, Pfizer, Inc., in collaboration with the World Heart Federation, started the “Heart Hero” campaign. Under this campaign, Pfizer, Inc. provides educational information and resources related to ATTR-CM to patients and physicians. Such campaigns are expected to boost the early diagnosis of the disease and increase the treatment rate over time.

The movement undertaken by key players to provide free-of-cost medicine under their CSR activities is also projected to fuel market growth. For instance, the Pfizer Patient Assistance Program provides Pfizer medicines such as VYNDAMAX free of cost to eligible patients. People diagnosed with amyloidosis can take advantage of such programs by enrolling at the Vandalink platform. Such programs are expected to encourage physicians to prescribe this drug to a patient as drug therapy in transthyretin amyloidosis patients.

Scope of The Report

Report Coverage	Details
Market Size in 2021	USD 4.87 billion
Revenue Forecast by 2030	USD 9.35 billion
Growth rate from 2022 to 2030	CAGR of 7.52%
Base Year	2021
Forecast Period	2022 to 2030
Segmentation	Type, therapy, disease type, distribution channel, region
Companies Covered	Pfizer, Inc.; Johnson & Johnson Services, Inc.; Ionis Pharmaceuticals, Inc.; Alnylam Pharmaceuticals, Inc.; BridgeBio Pharma, Inc.; Bristol-Myers Squibb Company; Acrotech Biopharma; AstraZeneca; Prothena; SOM Biotech

 

Key Drivers

• The global transthyretin amyloidosis treatment market is likely to be driven by an increase in the prevalence of disease, rise in the population of people of African origin, increase in awareness, improvement in diagnostic procedures, improvement of healthcare services, rapid economic growth in developing countries, and rise in research and development activity. On the other hand, less awareness in developing economies, misdiagnosis of the disease, high cost associated with disease diagnosis and treatment, limited clinical trials, and lack of efficient medication are major factors restraining the transthyretin amyloidosis treatment market.
• According to the Amyloidosis Foundation, there are roughly 126 different genetic variations in ATTR, with up to 53 types of genetic variations in non-transthyretin hereditary amyloidosis diseases. According to reports published on transthyretin amyloidosis, it has been estimated that nearly 10,000, or 1.1 per 100,000 individuals in the world are living with TTR-FAP. The age group of patients suffering from the disorder is between 30 and 40 years of age. It has also been observed that TTR-CM tends to affect older males aged 65 years and above. Familial amyloid polyneuropathy (TTR-FAP) leads to 100 different types of mutations in the transthyretin gene, which leads to protein misfolding. There is only a 50% chance of transferring the mutation to the next generation from an affected parent. On the other hand, the mutation that leads to familial amyloid cardiomyopathy is generally found in individuals of African origin. Amyloidosis related to age primarily affects Caucasian men who are aged 65 years and above.
• The diagnosis of transthyretin amyloidosis is carried out through tissue biopsy, genetic testing, and imaging studies of the heart. Currently, no efficient drugs for transthyretin amyloidosis are available in the market. A complete study of the family should be carried out In order to efficiently diagnose transthyretin amyloidosis. This is carried out through neurological assessment tests, which include small-fiber assessment, electrocardiogram, and laboratory tests.

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Type Insights

ATTR-CM dominated the market for transthyretin amyloidosis and accounted for a revenue share of more than 80.11% in 2021. This is owing to the high prevalence of disease and improving diagnosis rates at a significant pace. The exact prevalence of the disease is unknown, but various autopsy studies suggest that around 22% to 25% of people aged 80 years and above reported TTR amyloid deposition in the heart. Although in most cases, the degree of amyloid deposition is mild among ATTR-CM amyloidosis patients. Moreover, the launch of new drugs for the treatment of ATTR-CM is expected to boost segment growth. For instance, in May 2019, Pfizer Inc. received approval for its VYNDAQEL and VYNDAMAX from the U.S. FDA for the treatment of ATTR-CM.

ATTR-PN is estimated to register the highest growth rate over the forecast period. The growth of the segment is attributed to the high incidence rate and approval of new drug therapies for the treatment of ATTR-PN amyloidosis patients. Val30Met is the most common TTR mutation found in patients with neuropathy. Inotersen and Patisiran are the two FDA-approved drugs available for the treatment of ATTR-PN in the market. Both drugs inhibit the production of TTR in the liver and have shown to be effective in ATTR-PN. Furthermore, the fast-track approval of products worldwide is expected to boost segment growth. For instance, in June 2019, the Ministry of Health, Labor, and Welfare of Japan approved ONPATTRO (Patisiran) for the treatment of hATTR in adults.

Disease Type Insights

Hereditary transthyretin amyloidosis captured the largest revenue share of over 60.21% in 2021. Hereditary transthyretin-mediated amyloidosis is an autosomal dominant, rare, and fatal disease in which it impairs multiple organs, leading to death and disability. To overcome this situation, pharmaceutical companies are constantly working on drug development and several drugs are still in pipeline for commercialization. For instance, in August 2022, Attralus, Inc. received U.S. FDA authorization of orphan drug designation for 124I-AT-01, which could be used for the diagnosis of transthyretin amyloidosis.

Furthermore, several countries are anticipated to witness a higher number of cardiomyopathy cases. Therefore, intensive R&D is happening in the market for the proper diagnosis and treatment of the disease. For instance, in August 2022, Alnylam Pharmaceuticals, Inc. received FDA authorization for the drug Onpattro, which could be used for nerve pain treatment caused by hereditary transthyretin amyloidosis. Moreover, its phase 3 data shows that it has the potential to help patients suffering from cardiac-related issues for rare protein diseases.

Distribution Channel Insights

Hospital pharmacies captured the largest revenue share of over 30.08% in 2021. Most hospitals are working toward the diagnosis, research, and treatment of the disease. For instance, Brigham and Women’s Hospital in Massachusetts, U.S. focuses on diagnosing and treating misdiagnosed diseases. It has established the Amyloidosis Program, which offers access to innovative therapies, patient-centered care, and new clinical trials. The team of experts would educate physicians and patients and spread awareness among the patients. These initiatives would further contribute to the segment's growth.

Online pharmacies are anticipated to witness the fastest growth in the forecast period owing to the easy access of drugs to patients, which is also expanding the use to get health information. However, there is an increasing risk of patients purchasing products from illegal websites is expected to hamper the growth. For instance, an online publication stated that fraudulent online pharmacies are attempting to sell illegal generic versions of Vyndamax. This could be potentially unsafe for the customers. Therefore, promotional campaigns would further spread awareness among the public about safe and authentic online pharmacies, which could further prevent safety threats to patients.

Therapy Insights

The targeted therapy segment dominated the market and accounted for a revenue share of over 90.17% in 2021. Drugs such as Onpattro, Inotersen, and Tafamidis are included in this segment. Onpattro is administrated intravenously to ATTR-PN patients at an interval of 3 weeks with pretreatment of steroids and Benadryl to avoid side effects. Onpattro works by silencing the portion of RNA involved in producing TTR protein. Moreover, an increasing prevalence of the disease ATTR-PN and worldwide approval of these therapies are projected to fuel segment growth. For instance, in July 2018, the Food and Drug Administration (FDA) approved Onpattro (Patisiran) developed by Alnylam Pharmaceuticals, Inc. for the treatment of ATTR-PN in adults. Along with this, it holds the commercialization rights to Patisiran in Canada, the U.S., and Western European countries, while Sanofi Genzyme holds the right to the rest of the world.

Targeted therapy is expected to witness the fastest growth during the forecast period owing to increasing initiatives undertaken by key players to support the patients. For instance, Akcea Therapeutics, Inc. operates a patient assistance program “Akcea Connect” to support hATTR amyloidosis patients. Under this program, the eligible person and their families get free, private, and personalized support across the U.S. This will increase awareness about available drug therapies, encouraging general practitioners to prescribe these drugs to transthyretin amyloidosis patients. Hence, the high prescription rate and the high cost of these drugs are major factors fueling the targeted therapy segment growth.

Regional Insights

North America dominated the market with a revenue share of over 65.02% in 2021 and the region is anticipated to maintain its dominance during the forecast period. Increasing novel drugs prescription, proper reimbursement policies, and higher treatment rates in the region are factors supporting transthyretin amyloidosis treatment market growth in the region. The introduction of new therapies in the region is acting as a key market driver. For instance, in July 2019, Health Canada approved ONPATTRO for the treatment of hATTR with polyneuropathy in adults. This approval is projected to have a positive impact on the regional market.

The MEA region is projected to witness the fastest growth over the forecast period. The high unmet treatment needs, increasing prevalence of disease, and rising approval are expected to drive the market in the coming years. Moreover, major factors such as a large target population, good healthcare reforms, and an increase in disease awareness are expected to be the primary growth factors.

Key Players

• Pfizer Inc.
• Johnson & Johnson Services, Inc.
• Ionis Pharmaceuticals, Inc.
• Alnylam Pharmaceuticals, Inc.
• BridgeBio Pharma, Inc.
• Bristol-Myers Squibb Company
• Acrotech Biopharma
• AstraZeneca
• Astellas Pharma, Inc.
• Prothena
• SOM Biotech 

Market Segmentation

• By Type Outlook
  • ATTR-PN
  • ATTR-CM
• By Therapy Outlook
  • Targeted Therapy
    • Onpattro
    • Inotersen
    • Vyndaqel/Vyndamax
  • Supportive Therapy
  • Pipeline Therapy
• By Disease Type Outlook
  • Hereditary Transthyretin Amyloidosis
    • Polyneuropathy
    • Cardiomyopathy
    • Mixed Type
  • Wild Type Amyloidosis
• By Distribution Channel Outlook
  • Hospital Pharmacies
  • Specialty Pharmacies
  • Retail Pharmacies
  • Online Pharmacies
• By Regional Outlook
  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East and Africa (MEA)

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