Lomitapide
Treating Homozygous Familial Hypercholesterolemia (HoFH)
Homozygous familial hypercholesterolemia (HoFH) is a rare form of familial hypercholesterolemia (FH) that impacts roughly 1 in 300,000 people in the United States and worldwide. Someone with HoFH has inherited one FH gene from each of their parents.
This condition typically leads to untreated LDL cholesterol levels between 400 mg/dL and 1000 mg/dL – sometimes even higher. If cholesterol this high remains untreated, people with HoFH are at risk of developing premature heart disease, and most can’t lower their LDL enough with one, two, or even three cholesterol lowering medications.
In 2021, the Food and Drug Administration (FDA) approved lomitapide (brand name Juxtapid) for the treatment of people with HoFH only.
How does lomitapide work?
What makes lomitapide different, and particularly effective for HoFH patients, is that it does not require any functioning LDL receptors. This medication blocks the production of microsomal triglyceride transfer proteins (MTP) that help in the production of LDL cholesterol.
Studies have shown that lomitapide reduces LDL, on average, by 40%. This, on top of other lipid lowering medications, has helped people with HoFH lower their LDL levels.
