Transthyretin amyloidosis is a rare disease caused due to abnormal build-up of amyloidosis in the tissues of the body. The deposits of transthyretin amyloids cause illness due to damage in structure and function of the organs and moreover, they can affect any part of the body where deposits of transthyretin amyloids are present. The deposits mainly consists of abnormal, insoluble protein fibers called amyloid fibrils that are soluble in healthy people but when the protein is misfolded it becomes sticky and form a clump together, which is insoluble in nature and accumulate in the tissue.
Furthermore, there are around 30 different types of protein known to form amyloid, which result in different clinical conditions as different amyloidosis tend to affect different body organs. Transthyretin amyloidosis is progressive and rare fatal disease that destroys the nerve cells that governs the various functions of body. There are 2 types of transthyretin amyloidosis, namely, , transthyretin familial amyloid polyneuropathy and transthyretin familial amyloid cardiomyopathy. Among this, in transthyretin familial amyloid polyneuropathy nerves that control senses, movements, and involuntary bodily functions are affected. Whereas, in transthyretin familial amyloid cardiomyopathy the damage is caused to heart muscles and also impedes the heart’s ability to pump blood throughout the whole body.
Transthyretin Amyloidosis Treatment Market Taxonomy
By Disease Type
By End User
Rise in amount of transthyretin protein in the body expected to fuel the transthyretin amyloidosis treatment market
Transthyretin protein is formed in the body abundantly due to inherited transthyretin gene alteration that leads to transthyretin amyloids. According to Amyloidosis Foundation, there are approximately 126 different genetic variations in transthyretin amyloids. Transthyretin amyloids are not contagious but they are considered to be inherited as autosomal dominant disease that suggests a person needs to have a single copy of mutant gene. Each offspring’s have 50% chances of inheriting the gene from any one parent that suffers from transthyretin amyloids. As transthyretin amyloids leads to failure of any organ, it is important to treat the transthyretin amyloids, which is expected to favor the development of transthyretin amyloids treatment market.
Furthermore, according to Amyloidosis Foundation, medical statistics states that transthyretin amyloids is found approximately in the 4% of the U.S. African-American population which favors the growth of transthyretin amyloidosis treatment market.
Regional growth engines of the transthyretin amyloidosis treatment market
On the basis of regional segmentation, the transthyretin amyloids treatment market is segmented into North America, Europe, Asia Pacific, Latin America, Middle East and Africa. Moreover, most common variant in transthyretin amyloids are Val30Met, Thr60Ala, Leu58His, Ser77Tyr found in the U.S.. According to Amyloidosis Foundation, medical statistics states that the gene mutation for transthyretin amyloids is carried by approximately 1.5 million people in the U.S.. Whereas, tafamidis with trade name VYNDAQEL was first approved in Europe which is expected to favor the growth of transthyretin amyloidosis treatment market in Europe.
Furthermore, according to BioMed Central journal’s published data in March 2017, people from the Europe had highest number of TTR mutations, followed by the people from East Asia, the U.S., Central-South Asia and Africa, which is expected to favor the growth of transthyretin amyloidosis treatment market.
The key players dominating the transthyretin amyloidosis treatment market
Recently, in June 2017, the U.S. Food and Drug Administration FDA granted fast track designation to tafamidis which is the company’s investigational treatment for familial amyloid cardiomyopathy as there were no approved treatment in the U.S., manufacturers are innovating new treatment for transthyretin amyloidosis. The key players operating the market are Pfizer Inc., SOM Innovation Biotech, S.L., Valeant Pharmaceuticals International, Inc., Ionis Pharmaceuticals, Inc., and AstraZeneca plc.
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