Cholesterol Drug Juxtapid Costs $200K Plus, Has Serious Effects
Just in time for Christmas, the Food and Drug Administration (FDA) approved Juxtapid (lomidapide), a new cholesterol drug for individuals who have a rare genetic type of high cholesterol called homozygous familial hypercholesterolemia (HoFH). Juxtapid comes with a hefty price tag of $200K plus per year, and its own black box warning of serious side effects.
Who will benefit from Juxtapid?
Familial hypercholesterolemia (FH) is a genetic condition that is characterized by extremely high levels of total cholesterol and bad cholesterol: low-density lipoprotein (LDL). Two forms of FH exist: homozygous and heterozygous, which is associated with the types of genes that a person inherits and can pass on.
Without delving into the complexities of the genetics, suffice it to say that heterozygous FH occurs in about 1 in every 500 people around the world, while homozygous FH is much rarer, seen in about 1 in every one million people. Therefore, Juxtapid is a drug for a very limited number of individuals.
Children with HoFH are at risk of heart attack or coronary disease as young as one to two years old, so it is critical to effectively lower their LDL cholesterol as much as possible to slow progression of atherosclerosis (plaque accumulation in the blood vessels). According to Medscape, HoFH is associated with severe atherosclerosis that affects all the main arteries, including the carotid (arteries in the neck that supply the head), coronary (two major heart arteries), femoral (provides blood to the thighs, groin, lower abdomen), and iliac (pelvis and lower limbs).
In addition to cholesterol accumulations in the arteries, the substance also gathers under the skin, creating fatty deposits. These deposits may be identified as early as birth, which helps with early diagnosis and treatment of the disease.
Juxtapid for HoFH
According to Aergerion Pharmaceuticals, which makes Juxtapid, yearly treatment with the oral drug will cost $200,000 to $300,000, and along with the high price tag comes a black box warning of serious potential side effects for the liver. Specifically, use of Juxtapid can cause liver toxicity.
One way is through a rise in various substances in the liver, including alanine and aspartate aminotransferases, alkaline phosphatase, and total bilirubin. Levels of these substances must be checked regularly in people who take Juxtapid, and the drug should be discontinued if levels rise too high.
Juxtapid also can increase hepatic (liver) fat, resulting in a condition called hepatic steatosis, which is a risk factor for progressive liver disease. Hepatic steatosis can develop with or without increases in transaminases.
Because use of Juxtapid poses such a risk of liver toxicity, the drug is available only through a special program called the Juxtapid Risk Evaluation and Mitigation Strategy Program. Only certified doctors and pharmacies can prescribe and distribute the drug.
In addition to the risk of liver problems, the most common side effects (affecting 28% or more of users, based on clinical trials) are diarrhea, nausea, vomiting, heartburn, and abdominal pain.
Another drug, called Kynamro, made by Isis Pharmaceuticals, is awaiting FDA approval for HoFH. Unlike Juxtapid, Kynamro is an injectable drug, but it also is associated with toxic liver side effects.
Individuals with the rare disease HoFH have limited treatment choices. The new drug Juxtapid offers an option, although it comes with high costs in dollars and side effects.