Regional growth engines of the
familial amyloid cardiomyopathy treatment
On the basis of regional
segmentation, the Familial
Amyloid Cardiomyopathy Market can be segmented into North America, Europe,
Asia Pacific, Latin America, Middle East, and Africa. According to BioMed
Central journal’s published data in year March 2017, Europeans had highest
number of TTR mutations, followed by East Asians, American, Central-South
Asians, and Africans which favors the growth of familial amyloid cardiomyopathy
market.
Aggregation and deposition of
mutant and wild-type transthyretin protein (TTR) in heart results in familial
amyloid cardiomyopathy, which typically occur after age of 60. Familial amyloid
cardiomyopathy is also known as hereditary cardiac transthyretin amyloidosis or
hereditary amyloid cardiomyopathy. The protein transthyretin amyloid fibrils
infiltrate the myocardium that leads to diastolic dysfunction from restrictive
cardiomyopathy, which eventually result to heart failure. There are several
mutation in TTR which are associated with familial amyloid cardiomyopathy,
which includes V1221, P24S, V20I, A45T, Gly47Val, Glu51Gly, Gln92Lys, I68L, and
L111M. Most common mutation is V1221, which is substitution of isoleucine for valine
at position 122 that occurs in high frequency in African-Americans, which
favors the growth of familial amyloid cardiomyopathy treatment market.
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European Medicines Agency have
approved drug named tafamids (Vyndaqel) for slow progression of familial amyloid
cardiomyopathy in year 2013 which is expected to favor the market of familial
amyloid cardiomyopathy treatment. Most of the pharmaceuticals companies are
developing drugs for familial amyloid cardiomyopathy treatment, as it is
associated with progressive heart failure and it is universally fatal. Symptoms
for familial amyloid cardiomyopathy are weight loss, nausea, fatigue, dizziness
and collapse, palpitations, abnormal heart rhythms, disrupted sleep, and
angina. Genetic tests are recommended for detection of TTR gene.
Rise in amount of transthyretin
protein in the body fuels the familial amyloid cardiomyopathy treatment market
Transthyretin protein is formed
in the body abundantly due to inherited transthyretin gene alteration, which
leads to familial amyloid cardiomyopathy as gene associated transcribes the
protein. Gene alteration in body leads to alteration in sequence of body
proteins. Familial amyloid cardiomyopathy are inherited by autosomal dominant
allele, which can inherit the familial amyloid cardiomyopathy condition to
offsprings from one of the parent. Only one copy of mutated gene is sufficient
to cause the disease, which is expected to show growth in the familial amyloid
cardiomyopathy market. Familial amyloid cardiomyopathy is critical disease
leading to heart failure, which can favor the market. According to Amyloidosis
Foundation (AF), which provides early diagnosis and advanced treatment,
familial amyloid cardiomyopathy are rarely found with statistics estimating 1
per 100,000 population suffering from familial amyloid cardiomyopathy disease.
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The key players dominating the
familial amyloid cardiomyopathy market
Familial amyloid cardiomyopathy
not only affect lives of people but also affect upcoming generations, due to
which manufactures are in process of releasing the drugs, as there are very few
drugs approved for familial amyloid cardiomyopathy treatment. Recently in June
2017 the U.S. Food and Drug Administration (FDA), which provides accurate,
science-based health information to the public, granted fast track designation
to tafamidis the company’s investigational treatment for familial amyloid
cardiomyopathy as there were no approved treatment in the U.S. tafamidis with
trade name VYNDAQEL® was first approved in Europe. 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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