By Mary Welch

Genetics Institute Inc. said its biologics license application (BLA) in the U.S. and marketing authorization application in Europe for ReFacto Antihemophilic Factor, an albumin-free formulated recombinant Factor VIII, were accepted for review by the regulatory authorities.

If approved, Genetics Institute, of Cambridge, Mass., plans to sell ReFacto in North America. In Europe and elsewhere, the blood-clotting drug would be marketed by Wyeth-Ayerst, of Radnor, Pa., a division of American Home Products Corp. of Madison, N.J. American Home Products also owns Genetics Institute.

In addition, Genetics Institute formed the Genetics Institute Hemophilia Group, a division within the company dedicated to advancing care for people with hemophilia and their caregivers.

ReFacto is the first albumin-free recombinant product to seek licensing for hemophilia A, the most prevalent form of the bleeding disorder. Patients with hemophilia A either don't have or have a nonfunctioning Factor VIII gene, which along with Factor IX, produces clotting. Those without Factor IX (or whose Factor IX doesn't work) suffer from Hemophilia B — about one in every five victims.

Although recombinant Factor VIII products have been on the market since 1993, this is the first one free of albumin, which is added to the recombinant product after it is made, largely as a preservative.

Hemophiliacs Feel Like 'Canary In The Coal Mine'

"The question has always been if you have a recombinant product, why add albumin, which is a purified blood protein," said John Edwards, director for hemophilia products at Genetics Institute. "The reason people are preferring a recombinant product is for viral safety reasons. Then why add purified blood at the end? Even though the safety issues in receiving blood plasma have improved, the hemophilia community believes there is still the chance of getting other viruses — not just hepatitis or AIDS. They are very cautious. There are a lot of new viruses coming down. They feel like they're the canary in the coal mine."

If ReFacto is approved, Edwards said, it could grab a large share of the $1.5 billion hemophilia A market. Already half of the hemophilia market has switched to recombinant products.

In 1997, Genetics Institute began marketing BeneFix Coagulation Factor IX, the first recombinant Factor IX product, which also is albumin-free. The hemophilia B market is about $200 million worldwide and BeneFix posted sales of $50 million last year.

It is prescribed by more than 98 percent of all hemophilia treatment centers in this country. "The response was much quicker than we anticipated," Edwards said.

Genetics Institute purchased in August 1997 the development and commercialization rights to ReFacto from Pharmacia & Upjohn, of London, which will manufacture ReFacto for Genetics Institute as well as co-promote it in the Middle East and in the Nordic countries.

The Genetics Institute Hemophilia Group is the "culmination of [the company's] scientific interest and research for more than 15 years on hemophilia," said Edwards. "It reflects our scientific, clinical and now commercial interest in this area. It really formalizes our commitment to the hemophilia community. The group largely will work together to see what we can do to further help the hemophilia community and others with blood disorders. It will help us prioritize our actions as well as help build our existing community-based initiatives." *