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Data Insights Likelihood of Approval Analysis for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia)

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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 Hypercholesterolemia (Type II Hyperlipoproteinemia) overview

Familial hypercholesterolemia (FH) is a genetic disorder that affects the way the body processes cholesterol. People with FH have very high levels of low-density lipoprotein (LDL) cholesterol, also known as “bad” cholesterol, in their blood. This can lead to early and severe cardiovascular diseases, such as heart attacks and strokes. FH is caused by a mutation in one or more genes that are involved in removing LDL cholesterol from the blood. The most common gene affected is LDLR, which encodes the LDL receptor protein. Other genes that can cause FH are APOB, PCSK9, and LDLRAP1. FH is inherited in an autosomal dominant pattern, which means that having one copy of the mutated gene is enough to cause the condition. People who inherit two copies of the mutated gene have a more severe form of FH. FH affects about 1 in 250 people worldwide, but the prevalence may vary depending on the population and the genetic background. FH can be diagnosed by measuring the blood cholesterol levels, examining the physical signs of cholesterol deposits (such as xanthomas and arcus corneae), and performing genetic testing to confirm the mutation. FH can be treated with medications that lower cholesterol levels, such as statins, bile acid sequestrants, ezetimibe, and PCSK9 inhibitors. Some people with FH may also need other treatments, such as LDL apheresis or liver transplantation, to reduce their risk of complications. FH also requires lifestyle modifications, such as eating a healthy diet, exercising regularly, avoiding smoking, and managing other risk factors for cardiovascular diseases.

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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.