By Paul Elias
The Associated Press
For the past 20 years, Robert Cloud twice daily has ingested GHB, the notorious "date-rape" and rave-scene drug.
"It saved my law practice," said Cloud, a Cincinnati lawyer. "It saved me from disability."
GHB is illegal in the United States, classified alongside heroin and cocaine — except for Cloud and 725 other people who suffer from a rare but dangerous complication of the sleep disorder narcolepsy.
Orphan Medical, a tiny biotechnology company in Minnetonka, Minn., began selling the drug to those patients in October after receiving federal regulatory approval and agreeing to distribute it under some of the most severe restrictions ever imposed on a medicine.
Backed by government-sanctioned monopolies, a number of biotechnology companies are embracing similar niche markets as an alternative route to revenue.
Genzyme is one of the few profitable biotechnology companies thanks to its Gaucher's disease drug Cerezyme and Fabry's disease drug Fabrazyme. Another profitable Cambridge, Mass.-based company, Biogen, rang up about $1 billion in 2002 sales of Avonex, which treats multiple sclerosis.
These companies and others are developing therapies for such rare afflictions as hemophilia, the infant respiratory and heart ailment Pompe disease and an antivenin to treat rattlesnake bites. They see an opportunity to profit by avoiding competition while people with rare diseases are treated with cutting-edge medicines.
Orphan drug laws provide the motivation. Enacted a decade ago in the United States and three years ago in Europe, they encourage biotech firms to develop drugs for diseases that affect a small number of people who otherwise would be ignored as unprofitable markets. The U.S. law guarantees tax breaks, funding help and a seven-year monopoly. In Europe, the monopoly protection lasts a decade.
From 1983 to 2001, 228 orphan drugs were approved for sale in the United States, according to the Tufts Center for the Study of Drug Development. The center estimates that between 40 and 60 similar drugs were approved for sale in the country between 1963 and 1982, the year before the orphan drug law was enacted.
"The lack of competition is real nice," said Orphan Medical's Mark Perrin. The company has staked its financial life on treating rare diseases that most big drug companies snub as too small a market to serve.
Despite the regulatory hassles — only a single pharmacy is allowed to legally distribute it — and the tiny market, Orphan Medical has high hopes for the GHB drug.
In Cloud's case, Orphan Medical's GHB drug attacks cataplexy, a muscle weakness complication that can cause people to collapse without warning.
Martin Scharf, director of the Tri-State Sleep Disorders Center in Cincinnati, first gave GHB to Cloud in 1983 after reading about similar work being done in Canada.
Since then, Scharf has been a GHB Pied Piper of sorts, lobbying unsuccessfully against criminalizing the drug.
Throughout the 1990s, the government cracked down on illegal GHB use — it is abused as a party drug, sex and athletic enhancer and, because it can knock people out, a date-rape drug. Several dozen deaths are blamed on the chemical. But Scharf and others insist the drug, when properly administered by experts, holds great medicinal promises.
"This is an amazing story," Scharf said. He said GHB helps most cataplexy patients lead normal lives by cutting down on the attacks they suffer. Between 25,000 and 50,000 people suffer from cataplexy, and Orphan Medical hopes to sell its $5,000-a-year version of GHB to most of those narcoleptics.
Chief Executive John Howell Bullion told potential investors this week that he expects Orphan Medical to soon ring up $125 million in annual sales, enough to make it profitable.
Genzyme made a similar bet on treating Gaucher's disease, a genetic disorder that causes anemia and enlarged organs and can be deadly. Only 10,000 people suffer from the disease worldwide.
Genzyme's treatment, Cerezyme, won Food and Drug Administration approval in 1991 and Genzyme has been paid back handsomely for its risk: Half of its $1.08 billion in annual sales are from Cerezyme.
With only 3,500 patients taking Cerezyme, the drug still generated about $619 million in 2002. That works out to $170,000 per patient per year, making it one of the most expensive drugs on the market.
Insurance covers the cost of the drug for most patients. Genzyme provides the drug for free to those who don't have insurance or who can't afford it, chief executive Henri Termeer said.
The company's seven-year monopoly has long since expired. But its marketing position is so dominant Termeer sees little competition arising.
Termeer also said he is optimistic that an FDA advisory panel will approve the company's drug Fabrazyme, which has been approved in Europe.
About 40,000 men are stricken with Fabry's disease. Victims suffer from kidney failures, heart attacks and strokes and aren't expected to live past 40.
Termeer said Fabrazyme accounted for about $26 million in 2002 sales,
compared to $9 million in 2001. "The orphan drug law is one of the most
effective laws ever passed in the United States," he said.
Copyright © 2003 The Seattle Times Company