The Food and Drug Administration approval carves out one medical use for an otherwise illegal chemical.
Throughout the 1990s, the government had cracked down on illegal GHB use -- 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 GHB was hard to stop because it was easy for people to make by mixing up some common chemicals.
Now the maker of the only FDA-approved version, Orphan Medical Inc., will have to balance how to get GHB to the relatively few patients it could help while keeping it from falling into the wrong hands.
"No system, I believe, is foolproof, but there will be very close tabs" kept on every GHB shipment, said Dr. Russell Katz, FDA's neurologic drugs chief.
Narcolepsy is marked by recurring episodes in which patients suddenly fall asleep from a few seconds to an hour. GHB doesn't treat that symptom. However, anywhere from 20,000 to 50,000 narcolepsy patients also suffer from cataplexy, a muscle-weakness complication that can cause people to collapse without warning.
Orphan Medical's version of GHB, to be sold under the brand name Xyrem, marks the first FDA-approved treatment for cataplexy. Studies suggest Xyrem (pronounced Zy-rem) could reduce cataplexy attacks by up to 70 percent.
Originally developed as a surgical anesthetic, GHB was pulled off the market because of side effects: it depresses breathing and can cause coma, even kill.
Then in 1990, some companies began selling it as a dietary supplement, and use as a recreational drug, under such names as "Georgia homeboy," "cherry meth" and "liquid ecstasy," took off. Colorless and odorless, it made headlines when people slipped it into drinks, knocking out victims who often had no memory of what happened.
By the mid-90s, the government had declared any GHB use outside of FDA-sanctioned clinical trials illegal. A 2000 law toughened penalties so abusers or distributors could face a prison term. The Drug Enforcement Administration has blamed GHB for at least 58 deaths and 5,700 recorded overdoses since 1990.
Consequently, cataplexy patients who want to use Xyrem face a host of restrictions.
Every doctor who prescribes Xyrem must enroll on an FDA-monitored registry that also will record the name and medical progress of every patient who takes it.
Orphan Medical will hire one pharmacy to distribute Xyrem, sending it by Fed-Ex to the homes of properly registered patients who have certified they understand how to use it and the penalties for abuse.
The patient must sign for each shipment -- reports of lost or missing pills will immediately trigger an investigation.
Patients or doctors who divert the drug for illegal use can face jail, Katz warned.
Despite all the warnings, the FDA did conclude that Xyrem helps cataplexy, if taken twice a day.
However, it can cause the same breathing side effects in patients that abusers have had, the FDA cautioned -- especially if used with alcohol or other central nervous system depressants. Other side effects are rare but include bedwetting, sleepwalking and confusion.
The FDA will monitor the patient registry for reports of side effects, and urged doctors to report any patients' problems by calling 1-877-67-Xyrem.
Orphan Medical said it will begin sales by year's end but did not release a price.