Report Code: PP10135 | Report Type: Indication Pipeline Reports | Available format: |
Therapeutic Area(s): | Others |
Heterozygous familial hypercholesterolemia (HeFH) is an inherited genetic disorder that affects the body’s ability to control cholesterol. It is characterized by very high LDL (low density lipoprotein) cholesterol (above 190 for adults or above 160 for children) and family history of high cholesterol, heart disease or stroke. A very high level of LDL from birth leads to a twenty-fold increase in the risk of premature cardiovascular diseases. About 1 in 250 people around the world have HeFH and only about 10% of people with this medical condition have been diagnosed. Diagnosis is important because there are several early aggressive treatments available which can lower the risk for heart diseases or stroke.
The drug candidates of HeFH pipeline include, but not limited to, GEM-201 and anacetrapib. Some of the other companies having drugs in the HeFH pipeline includes Gemphire Therapeutics Inc. and Merck & Co. Inc.