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Richard N. Fogoros, M.D.

New Drug - Lomitapide - Approved For Severe Familial Hypercholesterolemia

By January 7, 2013

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Familial hypercholesterolemia (FH) is a genetic disease that produces very high levels of LDL cholesterol, and and premature coronary artery disease. FH is especially severe when the gene for FH is inherited from both parents, instead of just one - a condition referred to as homozygous FH.

Patients with homozygous FH have extremely high cholesterol levels, and often develop cardiovascular disease at a very young age. Their response to traditional cholesterol therapy (such as statins) is often insufficient. Some of them need to have regular treatments with apheresis (a procedure similar to hemodialysis) to lower their cholesterol.

Last week the Food and Drug Administration approved a new drug, called lomitapide (Juxtapid) for patients with homozygous FH. Lomitapide will be available only through a special program, by the direction of the FDA. These special precautions are necessary because the drug has been shown to produce liver disease in some patients. In fact, given the drug's propensity toward liver toxicity, the only reason the FDA approved it is that lomitapide offers a new therapeutic option to patients with this dangerous and difficult-to-treat condition. Without effective treatment, the risk of serious cardiovascular disease is very high in patients with homozygous FH.

Under the auspices of the FDA, the manufacturer of lomitapide (Aegerion Pharmaceuticals) has opened a support service for patients with homozygous FH and their families, which can help them determine whether this treatment is a reasonable consideration. The toll-free phone number for this new program is 1-85JUXTAPID (1-855-898-2743).

Comments
January 13, 2013 at 3:39 pm
(1) Robert Bramel says:

Many, many of us with heterozygous FH, perhaps 20-30 percent, have no disease process after a lifetime of high cholesterol. I’m 66, my LDL alone is 540, yet by the best techniques I have zero plaque and my heart functions as that of a 45 year old. From my teens I lived with “certain knowledge” that I would die in my forties and only found at age 55 that I was never ever at increased risk. My current risk, using EBCT, is in the lowest category, less than 1 in 500 of a heart attack.
The medical community does not concern itself with why so many of us should have no plaque buildup even though a little insight might be beneficial to a true understanding of the atherosclerotic process. I’ve volunteered multiple times to supposed researchers, but as one cardiologist said, “you represent such a small segment. Nobody cares about why.” She, of course, would have happily written me a prescription for a statin.

January 14, 2013 at 4:33 pm
(2) HeartHawk says:

Robert:

The Members would LOVE your story over at Track Your Plaque. They are all about using heart scans and advanced lipoprotein testing to determine true risk and not relying on cholesterol alone (lots of other factors). It’s a Members only site but I can get you a promo membership so you can post there if you drop me a line at hearthawk@wi.rr.com.

Regards,

HH

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