Market Insights
Amyloidosis is a rare disease caused by the buildup of amyloid, an abnormal protein that usually gets produced in bone marrow and deposits in any tissue or organ. According to the Amyloidosis Foundation, in the U.S., amyloidosis is the most common type, with approximately 4,500 new cases diagnosed every year. It usually affects people in the age range of 50–80; however, there are a few cases of people being diagnosed as early as their late 20s, and overall incidence statistics suggest that two-thirds of the patients are male. Blood and urine tests are carried out initially to diagnose amyloidosis, followed by an echocardiogram and imaging, tissue biopsy, bone marrow aspirate and biopsy, and organ biopsy.
The market experts suggest that, currently, there are no treatments available that can directly remove the amyloid deposits associated with amyloidosis. The treatment regime assigned for amyloidosis aims to prevent the further production of abnormal light chains while monitoring and treating any problems affecting the organs. There are several types of amyloidosis, such as A.L. (Amyloid Light Chain), A.A. (Amyloid Serum A Protein), hereditary (ATTR and Non-ATTR, TTR-Transthyretin Protein), wild-type ATTR, and others (Leukocyte Chemotactic Factor 2 [LECT2], Beta 2-microglobulin Amyloidosis [AB2M]).
Supportive care and type-specific treatment are the two treatment types practiced by medical practitioners. Supportive care treatment focuses on affected organs such as the kidney, heart, gastrointestinal (G.I.) tract, and peripheral nerves. Nephrotic syndrome and cardiomyopathy are treated with salt and fluid retention, accompanied by loop diuretics to restrict the ongoing protein loss. Kidney or heart transplantation is considered a preferred option when the disease is controlled at a specific level.
Medical experts suggest that to prevent recurrence in transplanted hearts, patients should be treated with aggressive anti plasma cell therapy with melphalan IV followed by an autologous stem cell transplant. The G.I. tract might develop diarrhea, which is controlled with the help of loperamide, and early satiety and gastric retention are treated with metoclopramide. Peripheral neuropathy is treated with gabapentin or pregabalin to relieve pain.
Chemotherapy is the most preferred option to treat amyloidosis, with drugs such as cyclophosphamide and melphalan combined with dexamethasone and prednisolone. The new drugs that are being evaluated for the treatment of amyloidosis are bortezomib, lenalidomide, and pomalidomide. Organ transplantations are carried out for the liver, kidney, and heart, but they are not appropriate for all patients, especially those with a late amount of amyloid deposition in their organs.
Dietary therapy is also used to reduce amyloid deposition in specific parts of the body, i.e., if amyloidosis affects the heart or kidneys, a low-sodium diet is recommended. In a few cases, stem cell transplantations or bone marrow transplants are also considered beneficial options; this type of stem cell transplantation is known as a peripheral blood stem cell transplant.
North America was observed as the largest market for amyloidosis therapeutic treatment, followed by Europe. In North America, rising awareness is associated with early disease diagnosis and the availability of accessible and developed healthcare infrastructure.
The epidemiology statistics suggest that primary systemic amyloidosis mainly occurs in around 8 million people per year, which comes to approximately 3,000 new patients annually in the U.S. In the U.K., the age-specific incidence rate is between 5.1 and 12.8 per million per year, with around 60 new cases annually.
The global amyloidosis therapeutic treatment market is segmented on the following basis:
Type of Treatment
- Supportive Care Therapy
- Furosemide
- Digoxin
- Amiodarone
- Loperamide
- Metoclopramide
- Gabapentin
- Pregabalin
- Chemotherapy
- Melphalan
- Cyclophosphamide
- Dexamethasone
- Prednisone
- Lenalidomide
- Pomalidomide
- Other Drugs
- Bortezomib
- Pipeline Analysis (Phase I, Phase II, and Phase III Clinical Trials)
- Projected sales of Phase III drugs are estimated to continue until 2023 (US$ Mn).
- IONIS-TTR (Ionis Pharmaceuticals, Inc. and GSK)
- NEOD001 (Prothena Therapeutics Ltd.)
- Patisiran/ALN-TTR02 (Alnylam Pharmaceuticals)
- Tafamidis (Pfizer, Inc.)
- Others
- Tabular Representation of Phase II and I Drugs
Geography
- North America
- Europe
- Germany
- France
- Italy
- U.K.
- Russia
- Rest of Europe
- Asia-Pacific
- India
- China
- Japan
- Rest of Asia-Pacific
- Latin America
- Brazil
- Mexico
- Rest of Latin America
- Middle East and Africa
- GCC Countries
- South Africa
- Rest of Middle East and Africa
Key players identified for the amyloidosis therapeutic treatment market include but are not limited to:
The key players in the market are Pfizer Inc., Bellus Health Inc., Astra Zeneca Plc, Merck & Co., GlaxoSmithKline, Abbott Laboratories, Inc., Takeda Pharmaceutical Company Ltd., Apotex, Inc., Celgene Corporation, Ionis Pharmaceuticals, Inc., Prothena Corporation Plc, Alnylam Pharmaceuticals, Inc., and others.
This report offers the following:
- An overview of the global markets for amyloidosis therapeutic treatment
- Market trends assessment for the period 2013-2023, with historical information for 2013, 2014, and 2015, and projections through 2023, with respective CAGRs during 2016-2023
- Qualitative assessment tools such as market drivers, challenges, and opportunities
- Market competition scrutiny tools such as market share analysis, fractal map assessment, and Porter’s Five Forces model
- Focus on each level of market segmentation based on product approvals, launches, and current and anticipated market dynamics.
- A general overview of the industry structure
- Company profiles highlight key information about the major players operating in the amyloidosis therapeutic treatment market.
