Genzyme Thinks Small, and Big, With Cholesterol-Lowering Drug Mipomersen

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Genzyme’s next step is to show mipomersen can help a broader group of patients—estimated at about 6,000 in the U.S.—who only have one bad copy of the cholesterol-clearing gene, but often have cholesterol scores so high that they need to visit a doctor regularly for what’s called an apheresis blood-filtering procedure. The procedure costs $75,000 to $150,000 a year, Butler says.

Then come the really big numbers. Last month, Genzyme said it started a pivotal trial of the drug for patients with a condition called heterozygous familiar hypercholesterolemia that also have one bad copy of the gene that contributes to their high cholesterol, but it isn’t so severe that they need the blood filtering. About 300,000 patients fall into that category, Butler says. Then there’s another 1 to 2 million patients who are either unable to tolerate statins, or are unable to get their cholesterol low enough by taking those drugs, he says. Two more clinical trials are expected to start later in the year to see if the drug can help those “high-risk” patients, Butler says.

Before Genzyme and Isis can market to that wide of a patient population, Genzyme will need results from what it calls an “outcome” study, Butler says. Genzyme hasn’t yet started the “outcomes” study or talked about its design, but generally the FDA wants to know whether drugs in the category can reduce the death rate and lower the number of heart attacks and strokes, Butler says.

All of this expansion naturally raises the question of price. How do you price a product that’s originally for just 300 seriously ill people (and therefore presumably pretty high), without making it too expensive to reach a mass market?

Butler didn’t want to discuss price with me, not yet anyway. “It’s way too early to talk about price. What drives price is the quality of the data,” he says.

He’s also not talking yet in hard numbers about how much impact mipomersen could have on Genzyme’s future bottom line, but the company has structured its partnership with Isis to prepare for big paydays to come. Genzyme and Isis plan to split profits from the drug, starting with a 70/30 split in favor of Genzyme, with a sliding scale that rises up to a 50/50 split once annual sales reach $2 billion. That might not mean much to a company like Pfizer, which is losing its patent on Lipitor in two years, but it means a lot to Genzyme, which had $3.8 billion in revenue last year as a company.

“This is a product that really fits Genzyme,” Butler says.

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