Xyrem, manufactured by Orphan Medical, has been approved for treating patients with narcolepsy who have episodes of cataplexy, a sudden loss of muscular control and weakness, usually triggered by emotions. The drug’s active ingredient is sodium oxybate, also known as gamma hydroxybutyrate (GHB).
Because of the abuse of GHB—it is used recreationally and in date rapes— distribution will be tightly controlled under Schedule III of the Controlled Substances Act. For the 20,000 to 50,000 people in the U.S. who suffer from narcolep-tic cataplexy, however, the news will be welcome, regardless of the restrictions. Cataplexy can cause a person with narcolepsy to collapse when the legs buckle. In clinical studies with 448 patients, the use of Xyrem, compared with placebo, reduced the number of cataplectic attacks.
Xyrem can have serious side effects, including confusion, depression, difficulty breathing while sleeping, loss of consciousness, and abnormal thinking. Abuse of the drug can lead to dependence. For these reasons, and in light of the drug’s history, the FDA and the manufacturer have designed a comprehensive risk management program (the Xyrem Success Program). For instance, prescribers and patients will be able to get the drug only through a single centralized pharmacy.