Hereditary Transthyretin Amyloidosis Market
Number of pages: 100 | Report Format: PDF | Published date: March 06, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
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Report Attribute |
Details |
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Market Size Value in 2022 |
US$ 1.43 billion |
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Revenue Forecast in 2031 |
US$ 2.46 billion |
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CAGR |
6.2% |
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Base Year For Estimation |
2021 |
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Forecast Period |
2023-2031 |
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Historical Year |
2021 |
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Segments Covered |
Drug Class, Distribution Channel, and Region |
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Regional Scope |
North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa |
According to the deep-dive market assessment study by Growth Plus Reports, the global hereditary transthyretin amyloidosis market was valued at US$ 1.43 billion in 2021 and is expected to register a revenue CAGR of 6.2% to reach US$ 2.46 billion by 2031.
Hereditary Transthyretin Amyloidosis Market Fundamentals
Hereditary transthyretin amyloidosis is a type of systemic amyloidosis disease caused by the inheritance of a gene mutation. The consequence of this hereditary mutation is an abnormally shaped amyloid protein. When these abnormal, misfolded amyloid proteins accumulate in the body’s nerves and other organs, they may harm tissue and organ activity. Despite having a gene mutation from birth, the harmful deposits typically do not appear until maturity. Although all forms of hereditary amyloidosis can result in severe complications, some people with this genetic mutation may never experience any disease symptoms. Others might experience a few minor health problems. Hereditary transthyretin amyloidosis symptoms differ based on the transthyretin genetic variant and the organ showing signs of amyloid deposition. The most common sites of amyloid deposits are the gastrointestinal tract and cardiac or nerve involvement, called cardiomyopathy and neuropathy. Other organs that may be affected include the kidneys, eyes, and carpal ligament, also known as carpal tunnel syndrome. Two strategies are followed for treatment: supportive treatment and source treatment.
While supportive care can differ depending on the disease mutation, organ transplantation has been successfully used when a kidney or heart is damaged. An organ transplant can considerably slow the progression of the disease, enhance the quality of life, and lengthen survival even if the generation of the variant amyloid protein persists. Stabilizers for transthyretin tetramers may help maintain the protein's regular circulating form. This prevents the protein from dissociating and undergoing conformational changes that lead to amyloid aggregation. Tafamidis and nonsteroidal anti-inflammatory drugs have been studied in controlled trials for their ability to function as transthyretin stabilizers in hereditary transthyretin amyloidosis. Tafamidis is an oral, kinetic transthyretin stabilizer that has received approval for treating hereditary transthyretin amyloidosis in adults with stage one symptomatic polyneuropathy in Europe, a few countries in Asia, and parts of South America.
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Hereditary Transthyretin Amyloidosis Market Dynamics
The prevalence of transthyretin amyloidosis is the most important factor driving the global hereditary transthyretin amyloidosis market revenue growth. Over the forecast period, the prevalence of hereditary transthyretin amyloidosis is expected to rise due to the increasing emergence of rare diseases in the global population. According to the National Center for Biotechnology Information, age-related transthyretin amyloidosis primarily affects the heart. Moreover, a significant risk factor for amyloidosis is aging.
Portugal, Sweden, and Japan are thought to be the only countries where the illness is endemic. It has been recorded in 36 countries worldwide, including the United States, with an estimated global prevalence of 5,000 to 10,000 people. According to the United Nations Department of Economic and Social Affairs, 727 million individuals were 65 or older worldwide in 2020. With a 16.0% CAGR, the global geriatric population is projected to hit 1.5 billion people by 2050. Furthermore, government and non-government organizations’ efforts to raise public knowledge of the disease and the advantages of its treatment are anticipated to increase the rate of early diagnosis and, consequently, the rate of treatment. For instance, the Australian Amyloidosis Network organized a biennial touring workshop in May 2021 to educate healthcare workers and patients with transthyretin amyloidosis about the condition. These workshops helped doctors and patients comprehend the complexity of the condition, which further aided in the creation of novel therapies for treating transthyretin amyloidosis, including hereditary transthyretin amyloidosis.
Numerous countries are expected to see an increase in cardiomyopathy cases. As a result, the industry is experiencing intense research and development for precise disease diagnosis and treatment. For instance, Alnylam Pharmaceuticals, Inc. obtained FDA approval for Onpattro in August 2022. Onpattro treats the nerve pain brought on by hereditary transthyretin amyloidosis. Additionally, according to phase 3 results, it may benefit individuals with rare protein disease-related cardiac problems also.
However, the high cost of the treatment is the major restraining factor for the global hereditary transthyretin amyloidosis market. Patients receiving tafamidis, patisiran, and inotersen treatment require more hospital visits for pre-treatment, administration, and monitoring, which results in higher patient burden and expenses. Most of the expenses go to drugs purchase, which cost an average of 291,076€ (for inotersen), 427,250€ (for patisiran), and 129,737€ (for tafamidis), per patient per year. Furthermore, in many countries, there is less knowledge of the disease. This factor has caused inaccurate medical counsel and delays in receiving necessary medical care. The hereditary transthyretin amyloidosis market is thought to be primarily constrained by shortcomings in clinical trials and false information about the disease.
Hereditary Transthyretin Amyloidosis Market Ecosystem
The global hereditary transthyretin amyloidosis market is analyzed from three perspectives: drug class, distribution channel, and region.
Hereditary Transthyretin Amyloidosis Market by Drug Class
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Based on the drug class, the global hereditary transthyretin amyloidosis market is segmented as RNAi agents (Patisiran), small interfering RNAs (siRNAs) (Vutrisiran), antisense oligonucleotides (Inotersen), transthyretin stabilizers (Tafamidis), and others.
The RNAi agents (Patisiran) segment accounted for the largest revenue share of the global hereditary transthyretin amyloidosis market in 2022. In August 2018, the U.S. Food and Drug Administration (FDA) and European Commission (EC) authorized the patisiran to treat adult polyneuropathy linked to hereditary transthyretin amyloidosis. Patisiran is a hepatocyte‐directed drug that utilizes endogenous mechanisms of RNAi to diminish spreading levels of abnormal and hereditary transthyretin‐mediated transthyretin protein. Patisiran is reported to enhance motor power, disability, functional mobility, nutritional condition, and autonomic symptoms compared to a placebo. Patisiran has received approval for treating hereditary transthyretin amyloidosis with polyneuropathy in more than 30 countries; the precise indications may vary with country and geographical area.
The market is anticipated to be driven by a strong and positive clinical pipeline. Several therapeutics are under development for the treatment of hereditary transthyretin amyloidosis. Also, the expanded research and development of new study methodologies for patisiran in hereditary transthyretin amyloidosis clear a novel therapeutic route. For instance, in September 2021, Alnylam Pharmaceuticals started the Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program to receive information on fetal/neonatal/infant outcomes, pregnancy consequences, and complications in women subjected to patisiran-LNP.
Tafamidis is a specific transthyretin stabilizer that inhibits the formation of amyloid and tetramer dissociation and is an effective first-line treatment for transthyretin amyloidosis, including the hereditary variant. Tafamidis has been shown to lower mortality and functional failure in patients with transthyretin amyloidosis. Moreover, several ongoing clinical studies on tafamidis as an effective treatment for hereditary transthyretin amyloidosis treatment significantly drive segment revenue growth.
Hereditary Transthyretin Amyloidosis Market by Distribution Channel
Based on the distribution channel, the global hereditary transthyretin amyloidosis market is segmented into hospital pharmacies, retail pharmacies, and online pharmacies.
The hospital pharmacies segment accounted for the largest revenue share of the global hereditary transthyretin amyloidosis market in 2022. Hospital pharmacies maximize patient care by working with doctors, nurses, and other healthcare professionals to provide the proper medications and supplies according to the set treatment protocols. The American Society of Health-System Pharmacists surveyed hospital pharmacy practice nationally. The results showed that hospital pharmacists perform better in patient care and offer more medication-related patient transition-of-care interventions, 24/7 medication order reviews, and counseling. The hospital pharmacy has a big impact on the economics of health expenditures. The country’s socioeconomic development and its citizens’ well-being are directly impacted by health. A hospital pharmacy provides patients access to medications, equipment, and other supplies needed for injury, illness, disease diagnosis, mitigation, prevention, and therapy. Hospital pharmacies acquire, stockpile, and supply medications for delivery, compounding, and pharmaceutical care. From the patient’s standpoint, hospital pharmacy services offer drugs, knowledge, and guidance to both inpatients and outpatients, as well as to the medical staff and others providing care to the patients.
Several government programs, such as subsidized medications, assist patients in purchasing expensive medications for rare illnesses, regardless of their ability to pay. Therefore, the increasing drug costs and unavailability of medicines in retail pharmacies influence the patient to prefer hospital pharmacies, leading to a significant increase in revenue. For instance, the Government of India launched National Policy for Rare Diseases (NPRD) in March 2021 to treat rare disease patients and provide financial support of up to INR 50 lakhs to patients suffering from any category of rare diseases and for treatment in any of the Centre of Excellence mentioned in NPRD-2021, outside the Umbrella Scheme of Rashtriya Arogaya Nidhi.
Hereditary Transthyretin Amyloidosis Market by Region
Based on the region, the global hereditary transthyretin amyloidosis market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa.
North America accounted for the largest revenue share of the global hereditary transthyretin amyloidosis market in 2022. The market for hereditary transthyretin amyloidosis In North America is likely to be influenced by factors such as a rise in disease prevalence, an increase in the number of people of American ancestry, a rise in disease awareness, advancements in diagnostic techniques, enhancements in healthcare services, accelerated economic developments, and increase in research and development activities. North America dominates the global hereditary transthyretin amyloidosis market due to a larger patient pool, an increase in the number of diagnostic procedures, the existence of top manufacturers, and quick drug development. Nearly 4,500 new cases of transthyretin amyloidosis are identified in the United States each year, according to the Amyloidosis Foundation.
Asia Pacific is expected to offer lucrative opportunities to the hereditary transthyretin amyloidosis market. This can be attributed to an increase in demand for supportive care therapeutics due to increased healthcare costs in the region, a rise in medical tourism, augmentation of the pharmaceutical industry, and a high acceptance rate for novel products.
Hereditary Transthyretin Amyloidosis Market Competitive Landscape
Several industry participants are trying to develop and commercialize medications to diagnose and treat transthyretin amyloidosis accurately. For instance, the FDA designated Eplontersen of AstraZeneca plc, which treats transthyretin-mediated amyloidosis, as an Orphan Drug Designation in the United States in January 2022.
The prominent companies holding the larger revenue share of the global hereditary transthyretin amyloidosis market are:
Hereditary Transthyretin Amyloidosis Market Strategic Developments
Hereditary transthyretin amyloidosis is a rare genetic disorder caused by the inheritance of a gene mutation for creating misfolded amyloid proteins.
A rise in the development of new drugs and treatments is expected to drive the global hereditary transthyretin amyloidosis market significantly in the near future.
Alnylam Pharmaceuticals, Inc., Akcea Therapeutics, PTC Therapeutics, Swedish Orphan Biovitrum, and Pfizer Inc. are among the top market players.
The global hereditary transthyretin amyloidosis market is expected to grow at a revenue CAGR of 6.2% during the forecast period from 2023 to 2031.
*Insights on financial performance are subject to the availability of information in the public domain