By Lisa Seachrist

Novo Nordisk A/S received marketing approval for its recombinant version of coagulation Factor VIIa, NovoSeven, for the treatment of bleeding episodes in hemophiliacs whose bodies reject other coagulation treatments.

NovoSeven is already approved in Europe, and was recently approved in Canada. In the U.S., the treatment has been available for compassionate use. After appropriate labeling changes, Denmark-based Novo Nordisk will make the drug available in the U.S. in three weeks.

¿It¿s bad enough to have hemophilia; then, to have your body reject the drugs to stop bleeds is really sad,¿ said Elaine Metcalf, spokesperson for Novo Nordisk. ¿NovoSeven gets around that problem.¿

For most people, a cut or injury results in bruising or bleeding that is squelched by a number of coagulation factors. About 25,000 men and boys in the U.S. with hemophilia aren¿t so lucky. Their bodies fail to produce one or more of the dozen or so proteins that cause coagulation.

Patients Given Relief In Hours

As a result, a cut or bruise requires immediate medical attention to infuse the missing coagulation factors into a hemophiliac¿s bloodstream, and thereby stymie the uncontrolled and painful bleeds. Eighty percent of hemophiliacs with hemophilia A need Factor VII. The remaining 20 percent with hemophilia B require Factor IX.

The problem is that, as hemophiliacs continue to receive the appropriate factor to permit their blood to clot, about 20 percent begin to develop antibodies to the coagulation factors that are essentially foreign to their bodies. These antibodies, known as inhibitors, block the action of the coagulation factors.

Patients with inhibitors need to be hospitalized for weeks at a time, while physicians pump coagulation factors into their bloodstreams, in the hopes of swamping the inhibitory antibodies to stimulate clotting.

NovoSeven does an end run around this problem by promoting clotting independently of either Factor VIII or Factor IX. A recombinant version of the coagulation Factor VIIa, NovoSeven binds to tissue factor, a protein that is expressed in the damaged vessel wall. This complex leads to activation of the coagulation system and clot formation at the site of injury.

Harold Roberts, professor of medicine at the University of North Carolina, in Chapel Hill, participated in clinical trials of NovoSeven.

¿With severe hemophilia, you have an inhibitor that prevents the use of the correct factor,¿ he said. ¿You¿ve already got a bad disease, and then you can¿t use the drugs to treat it. With NovoSeven, patients get relief within hours.¿

Metcalf noted patients can get a response to the correct dosage within minutes, depending upon the patient.

¿One of the things I really like about this product is the fact that it¿s recombinant,¿ Roberts said. ¿I certainly prefer giving a patient a recombinant product over something pooled from the plasma of 20,000 people.¿

NovoSeven will cost $1.40 per microgram, with dosing dependent upon the patient¿s weight. Metcalf said the price is comparable to other hemophilia drugs.

Novo Nordisk also is testing the drug¿s usefulness in liver transplantation and preventing restenosis of arteries, following balloon angioplasty.

Novo Nordisk¿s stock (NYSE:NVO) closed Friday at $51.50, up $1.5625.