FDA Advisory Committee Finds Xyrem® Effective for Treating Cataplexy

Minneapolis, MN - June 6, 2001

The Peripheral and Central Nervous System Advisory Committee of the U.S. Food and Drug Administration (FDA) voted that Orphan Medical's Xyrem (sodium oxybate) oral solution is an effective treatment for cataplexy associated with narcolepsy.

The Advisory Committee voted 6 to 3 that Xyrem is effective in treating cataplexy at a dose of 9 grams per day. It voted 5 to 4 that it is also effective at a dose of 6 grams per day. The active ingredient in Xyrem is gamma hydroxybutyric acid (GHB). On the issue of safety, the committee voted 4 to 4, with one abstention, and voted 8 to 1 that a risk management plan is necessary for the safe use of Xyrem, as Orphan Medical had recommended.

"We are encouraged by the outcome of the committee's deliberations, even though the voting was not unanimous, and we're pleased by the committee's general view that new therapies are needed for cataplexy associated with narcolepsy. We will meet promptly with the FDA and feel confident that we can address the clinical issues discussed by the committee," said John Bullion, CEO of Orphan Medical. "Xyrem is now one step closer to approval and helping physicians better manage narcoleptic patients with cataplexy. We remain committed to the value of this drug in an under-served population."

In addition to the clinical issues raised by the committee, Orphan Medical will also address with the FDA issues regarding product labeling, the proposed distribution system (to assure safe use), and manufacturing controls to assure compliance with FDA's Good Manufacturing Practice regulations.

Xyrem was designated by the FDA for priority review in October 2000. The Advisory Committee meeting is one of the last stages in the approval process. If approved, Xyrem would be the only treatment approved by the FDA as effective in managing cataplexy in those afflicted with narcolepsy.