Familial Amyloid Cardiomyopathy is an indication for drug development with over 10 pipeline drugs currently active. According to GlobalData, preregistered drugs for Familial Amyloid Cardiomyopathy have a 66.67% likelihood of approval (LoA) indication benchmark. GlobalData’s report assesses how phase transition success rate (PTSR) and likelihood of approval (LoA) scores for pipeline drugs in Familial Amyloid Cardiomyopathy compared to historical benchmarks. Buy the report here.

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GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Familial Amyloid Cardiomyopathy overview

Familial amyloid cardiomyopathy (FAC), or transthyretin amyloid cardiomyopathy (ATTR-CM), results from the aggregation and deposition of mutant and wild-type transthyretin (TTR) protein in the heart. TTR is usually circulated as a homo-tetramer; however, in FAC patients, TTR dissociates and misassembles into amyloid fibrils that are insoluble and resistant to degradation. Due to this resistance to degradation, when amyloid fibrils accumulate in the heart’s walls, specifically the left ventricle, causing rigidity that prevents the heart from properly relaxing and refilling with blood. This is called diastolic dysfunction and can further lead to heart failure.

For a complete picture of PTSR and LoA scores for drugs in Familial Amyloid Cardiomyopathy, buy the report here.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article. 

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.