By Charles Craig

Genetics Institute Inc. achieved a milestone Wednesday when the FDA approved its hemophilia B drug, BeneFix, which is recombinant Factor IX protein. It is the first product brought to market in the U.S. by the 16-year-old company.

BeneFix won approval two months after the Cambridge, Mass., biotechnology firm was acquired by its majority stockholder, American Home Products Corp., of Madison, N.J., for more than $1 billion.

Genetics Institute has created other drugs that were licensed to pharmaceutical firms for commercialization: Factor VIII for hemophilia A; erythropoietin (EPO) for anemia; and granulocyte-macrophage-colony-stimulating factor (GM-CSF) for restoring white blood cells depleted by cancer chemotherapy, bone marrow disorders and antiviral treatments.

But submission of a new drug application (NDA) for BeneFix in September 1996 marked Genetics Institute's first FDA filing. The second came after the American Home Products takeover in late December 1996, when Genetics Institute submitted an NDA for Neumega, recombinant interleukin-11, for boosting platelet growth in cancer chemotherapy patients and others with bone marrow disorders.

Genetics Institute, operating as a subsidiary of American Home Products, will sell BeneFix in North America with its own sales force. The drug is under review in Canada and Europe. Baxter Healthcare, of Deerfield, Ill., licensed BeneFix for European sales.

Dennis Harp, spokesman for Genetics Institute, said BeneFix will be available for U.S. patients in two days.

Hemophilia B strikes mostly males and is caused by a deficiency in the Factor IX blood clotting protein. The genetic disease, resulting in uncontrolled bleeding, is less common than hemophilia A, which afflicts about 50,000 people and is caused by a deficiency of Factor VIII, another blood clotting protein.

About 3,000 people in the U.S. are affected by hemophilia B and another 4,000 suffer from the disease in Canada, Europe and Japan.

Harp said BeneFix's price will be equivalent to that of Factor VIII, which is sold by Baxter in North America and Europe under the brand name Recombinate.

The annual average cost per patient is $25,000, but that figure includes those who only need the drug when undergoing surgery, Harp said. The cost for patients who require frequent treatments is about $100,000 a year.

Prior to BeneFix's approval, hemophilia B patients had to rely on blood-derived Factor IX, which carries with it the potential risk of viral contamination from donors.

Harp said BeneFix's cost is about seven percent higher than the blood-derived product.

U.S. market clearance of BeneFix followed by two months a unanimous recommendation for approval by an FDA advisory committee. (See BioWorld Today, Dec. 13, 1996, p. 1.)

As for the other two Genetics Institute products on the market, Boehringer Mannheim GmbH, of Mannheim, Germany, and Chugai Pharmaceutical Co. Ltd., of Tokyo, sell EPO; and Novartis AG, of Basel, Switzerland, and Schering-Plough Corp., of Madsion, N.J., sell GM-CSF. *