The global amyloidosis treatment market size was valued at USD 3.6 billion in 2017 and is anticipated to exhibit a CAGR of 7.2% over the forecast period. Increased adoption of novel targeted therapies and transplantation, the emergence of biologics, and the rising geriatric population are some of the factors that will collectively drive the market.
Amyloidosis is a group of rare diseases characterized by the buildup of amyloids (aggregates of proteins) that are folded in an incorrect way and get deposited in several organs. The most commonly affected organs include the heart, kidneys, liver, soft tissue, peripheral nervous system, and the gastrointestinal tract.
Organ involvement can be different among patients; however, the reason for this difference remains unknown. Amyloidosis can be categorized as either localized or systemic, based on if it only gathers in a specific part of the body or if it is simultaneously located in different parts of the body. AL, AA, and ATTR amyloidosis are the most common systemic variants of this disease.
In the U.S., AL amyloidosis is the most common type, with approximately 3,900 new cases diagnosed every year. The median age of diagnosis for this disease variant is mid-60. Its annual incidence is about 14 cases per million people in western countries, which is approximately one-fifth of that of multiple myeloma, a blood cancer caused by clonal plasma cells in the bone marrow. Furthermore, symptoms such as weight loss, fatigue, edema, and paresthesia often overlap with other common diseases, making its diagnosis difficult.
AL amyloidosis is associated with a high mortality rate of approximately 35% within one year of diagnosis, with a median survival between 12 and 18 months. The overall survival in this disease is poorer than multiple myeloma, which shares certain similarities with AL amyloidosis in relation to both pathogenesis and treatment options.
Currently, available amyloidosis treatments provide symptomatic relief and palliative care. These include chemotherapy, targeted therapy, supportive care, surgery, and stem cell transplantation. High-dose chemotherapy followed by autologous stem cell transplant remains the preferred therapy for low-risk patients in the U.S. due to the high rate of hematological remission and long-term disease control following treatment.
Targeted therapies and stem cell transplants are gaining traction in developed countries. However, the majority of the world’s population does not have access to sufficient healthcare infrastructure to ensure rapid diagnosis and treatment in low-income economies. Additionally, one of the major challenges for the amyloidosis treatment market is the high cost of these therapies, which creates a financial burden for patients based in underdeveloped regions. Targeted therapy is likely to demonstrate the fastest growth in the global market, followed by stem cell transplant and surgery. It is expected that chemotherapy and supportive care will lose market share over the forecast period due to adverse side effects and lower efficacy.
U.S. dominated the global market with a share of more than 27.0% in 2017, followed by France and U.K. Although France is projected to exhibit the highest CAGR among the seven major markets, the U.S. is expected to retain its market lead throughout the forecast period. The potential launch of pipeline drugs increased the adoption of novel therapeutics, and the high cost of treatment in the U.S. is a few factors responsible for its dominant share.
EU5 countries together accounted for more than 60.0% of the market in 2017. This is owing to the fact that two of the three late-stage drugs have been approved for amyloidosis treatment in Europe. Emerging markets for amyloidosis such as China, Sweden, Portugal, Turkey, South Africa, Brazil, Mexico, Saudi Arabia, and India are expected to show attractive growth over the forecast period due to encouraging government policies, increased investment, and improved healthcare infrastructure. The large population base in these countries with low per capita income has resulted in high demand for affordable healthcare, which will drive the rise of chemotherapy generics in this market. There is a critical need for curative treatment options with improved efficacy.
Key players operating in the market include Pfizer, Inc.; Ionis Pharmaceuticals, Inc.; Takeda Pharmaceutical Company Ltd.; Johnson and Johnson Pvt. Ltd.; and Alnylam Pharmaceuticals, Inc. Alnylam and Ionis are projected to dominate the competitive landscape post the worldwide approval of their pipeline candidates - Tegsedi and Onpattro, respectively.
Pfizer also holds the promise of gaining significant market share by 2025, supported by strong sales of Vyndaqel. Late-stage pipeline drugs such as Johnson & Johnson’s Darzalex and Takeda’s Ninlaro have demonstrated favorable safety and efficacy results. Some of the other companies actively investing in R&D of amyloidosis treatment are GlaxoSmithKline Pharmaceuticals Ltd.; Celgene Corp; Eidos Therapeutics, Inc.; Corino Therapeutics, Inc.; Spectrum Pharmaceuticals, Inc.; Prothena Corporation plc; Caelum Biosciences, Inc.; Proclara Biosciences Inc.; Arcturus Therapeutics Ltd.; and Neurimmune Holding AG. In the near future, the market will witness the introduction of biologics such as GSK2398852, Darzalex, PRX 004, CAEL101, LUNAR-101, and NI 301.
Base year for estimation
2018 - 2025
Revenue in USD Million & CAGR from 2017 to 2025
U.S., Japan, U.K., Germany, France, Spain, Italy
Revenue forecast, company share, competitive landscape, growth factors, and trends
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This report forecasts revenue growth at global, regional, and country levels and provides an analysis on the latest industry trends in each of the sub-segments from 2017 to 2025. For the purpose of this study, Grand View Research, Inc. has segmented the global amyloidosis treatment market report based on treatment type and country:
Treatment Outlook (Revenue, USD Million, 2017 - 2025)
Stem Cell Transplant
Country Outlook (Revenue, USD Million, 2017 - 2025)
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