28 June 2016 - No more than a few hundred American children have a rare disease characterized by ultrahigh levels of bad cholesterol.

Yet to the giant drug maker AstraZeneca, this small group could be worth billions of dollars.

The company is making a bold attempt to fend off impending generic competition to its best-selling drug, the anti-cholesterol pill Crestor, by getting it approved to treat the rare disease. In an unusual legal argument, the company says Crestor is entitled to seven years of additional market exclusivity under the Orphan Drug Act, a three-decade-old law that encourages pharmaceutical companies to develop treatments for rare diseases.

Critics say AstraZeneca is trying to abuse the law, since the overwhelming use of Crestor is for treating adults with high cholesterol, not children with the rare disease.

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