Transthyretin amyloid cardiomyopathy is a rare inherited and slowly progressive cardiac disease, in which a protein-based infiltrate deposits in tissues as beta-pleated sheets. The disease is characterized by an abnormal buildup of deposits of a mutant and wild-type transthyretin (TTR) protein in the heart called TTR amyloid fibrils in the body's organs and tissues. This leads to diastolic dysfunction from restrictive cardiomyopathy and eventual heart failure. In the U.S., the most common mutation (V122I) is seen predominantly among individuals of African descent. There are three types of transthyretin amyloid cardiomyopathy: light-chain amyloidosis, mutant transthyretin amyloidosis, and wild-type transthyretin amyloidosis. All three forms of transthyretin amyloid cardiomyopathy are progressive and fatal. For patients with wild-type transthyretin amyloidosis cardiomyopathy and mutant transthyretin amyloidosis cardiomyopathy, symptoms usually manifest later in life (after the age of 50). The median period of survival is three to five years from the time of diagnosis.
The global transthyretin amyloid cardiomyopathy therapeutics market is estimated to expand at a rapid pace in the coming years. A rise in the prevalence of transthyretin amyloid cardiomyopathy, increase in research and development, introduction of novel disease therapies, and rise in demand for effective therapies among patients are projected to fuel the growth of the global transthyretin amyloid cardiomyopathy therapeutics market. In February 2019, Eidos Therapeutics, Inc. initiated its ATTRibute-CM, a Phase 3 study of AG10 in patients with transthyretin amyloid cardiomyopathy. The design of the ATTRibute-CM study, which incorporates feedback from the FDA, includes two potentially registrational endpoints. The prevalence of transthyretin amyloid cardiomyopathy is highly variable among countries across the globe. Despite inherent uncertainty, studies suggests that the prevalence of transthyretin amyloid cardiomyopathy is underestimated, particularly for the mutant transthyretin amyloid cardiomyopathy subgroup. A rise in the prevalence of cardiovascular diseases and increase in health care insurance coverage are other factors likely to accelerate the growth of the global transthyretin amyloid cardiomyopathy therapeutics market. According to the World Health Organization, cardiovascular diseases accounted for 17.7 million deaths in 2015, representing 31% of all global deaths. However, stricter regulation for product approvals and high product cost are likely to restrain the transthyretin amyloid cardiomyopathy therapeutics market.
The transthyretin amyloid cardiomyopathy therapeutics market can be segmented based on treatment, disease, end-user, and region. In terms of treatment, the market can be classified into drug therapy and medical devices. The drug therapy segment can be sub-segmented into symptom-directed treatment and slow progression drugs. Based on disease, the transthyretin amyloid cardiomyopathy therapeutics market can be classified into wild type transthyretin amyloidosis and mutant transthyretin amyloidosis. In terms of end-user, the market can be categorized into hospital pharmacies, retail pharmacies, and online pharmacies.
Based on region, the transthyretin amyloid cardiomyopathy therapeutics market can be segmented into North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America dominates the global transthyretin amyloid cardiomyopathy therapeutics market due to new product innovation, high health care expenditure, and government awareness programs. In January 2019, the U.S. Food and Drug Administration (FDA) accepted the filing of Pfizer Inc.’s New Drug Applications (NDAs) for tafamidis for the treatment of transthyretin amyloid cardiomyopathy. Pfizer has submitted two NDAs based on two forms of tafamidis: meglumine salt and free acid. Europe is a prominent region of the transthyretin amyloid cardiomyopathy therapeutics market. The market in Asia Pacific is expected to grow at a higher rate due to a rapid rise in the population, rise in the prevalence of chronic diseases, increase in health care coverage, and rise in investment in research and development. Emerging regions such as Latin America and Middle East & Africa are projected to offer growth opportunities to the global transthyretin amyloid cardiomyopathy therapeutics market due to low awareness among people.
Major players operating in the global transthyretin amyloid cardiomyopathy therapeutics market include Pfizer, Inc., Ionis Pharmaceuticals Inc., Ltd., Eidos Therapeutics, Inc., Alnylam Pharmaceuticals, Prothena Corporation plc, and Regeneron Pharmaceuticals, Inc. Approvals and launches of novel therapies for the treatment of transthyretin amyloid cardiomyopathy are expected to be a major factor driving the market growth over the forecast period. For instance, the introduction of RNAi Therapeutics for transthyretin amyloid cardiomyopathy by Alnylam Pharmaceuticals. A robust pipeline of drugs is estimated to boost the transthyretin amyloid cardiomyopathy therapeutics market during the forecast period.
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