Toddlers Facing Heart Attacks Await Ruling on New Drugs

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http://www.bloomberg.com/news/2012-10-08/toddlers-facing-heart-attacks-await-ruling-on-new-drugs.html


Christian Jacobs, 20, was diagnosed with homozygous familial hypercholesterolemia, a rare genetic disease, at age 2. It led his cholesterol levels to be 4 times higher that of a healthy adult. Photographer: Chris Kirzeder/Genzyme via Bloomberg

Like any patient at risk of a heart attack, Kennedi Thompson has been counseled by her doctor to eat a low-fat diet and take prescription statins to reduce her cholesterol. Unlike most others facing heart disease, Kennedi is 3 years old.

She has a rare genetic disorder that has pushed her cholesterol level to four times that of a healthy adult. If left untreated, the disease -- called homozygous familial hypercholesterolemia, or HoFH -- can spur a patient’s first heart attack or stroke in their teens, give them the arteries of an octogenarian by age 20, and kill by the time they’re 30.

The estimated 3,000 Americans with HoFH may get new hope next week. Advisers to the U.S. Food and Drug Administration meet Oct. 17 and Oct. 18 to debate the risks and benefits of two experimental drugs for the disease, from Aegerion Pharmaceuticals Inc. (AEGR) and Isis Pharmaceuticals Inc. (ISIS), partnered with Sanofi’s Genzyme unit. The advisory panels’ recommendations often influence approval decisions.

“I’m crossing my fingers, crossing my toes, crossing everything that they get passed,” said Christian Jacobs, a 20- year-old college student who was diagnosed with HoFH at 2 and last week was preparing to have stents implanted to unclog his arteries.