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Help For Hemophiliacs

Hemophilia patients have won a new, genetically engineered drug called NovoSeven that may offer significant help when standard therapy fails.

In a person with Hemophilia blood doesn't clot properly, so they are at risk of hemorrhage from even minor injuries. They are treated with infusions of blood clotting factors called Factor 8 or Factor 9.

But about 10 percent of hemophiliacs those treatments eventually lose their ability to work. The patient's body develops "inhibitors" that block them. Manufacturer Novo Nordisk Pharmaceuticals estimates that NovoSeven could help such people.

Doctors have tried giving patients massive amounts of Factor 8, an expensive endeavor that sometimes works by overwhelming the inhibitors said Food and Drug Administration hematology chief Dr. Mark Weinstein. A few other drugs offer mixes of different clotting factors, but too often patients don't respond.

NovoSeven, which the FDA quietly approved last Friday, offers the first alternative. Also called recombinant Factor 7a, it was genetically engineered to contain no human proteins. The drug bypasses the need for typical hemophilia blood factors, instead targeting proteins in the damaged blood-vessel wall to clot blood, Weinstein explained.

The drug carries an average wholesale price of $1.40 per microgram. Novo Nordisk chose an unusual pricing method because patients' doses are customized according to body weight and the severity of bleeding. A 132-pound, moderately bleeding adult, for example, might require two 5,400-microgram doses, roughly $7,500 per dose.