Positive Phase III Results for Sodium Oxybate in Fibromyalgia Syndrome announced

Jazz Pharmaceuticals, Inc. and UCB today announced positive preliminary top-line results from the first of two Phase III pivotal clinical trials of sodium oxybate (JZP-6) for the treatment of Fibromyalgia Syndrome (Fibro).

The randomized, double-blind, placebo-controlled study achieved its primary endpoints, demonstrating that sodium oxybate significantly decreased pain and fatigue, and improved daily function, in patients with Fibro.

The study's lead investigator, I. Jon Russell MD, PhD, said today that:

"There is a significant unmet need in diagnosing and treating millions of patients with fibromyalgia. Potential new treatments that address the various symptoms can have a significant impact on patients' quality of life"

Dr Russell is also Associate Professor of Medicine, Division of Clinical Immunology and Rheumatology, and Director, University Clinical Research Center, University of Texas Health Science Center at San Antonio.

The 14-week study included 548 adult patients with Fibro, who were randomly assigned to either one of two doses of sodium oxybate or placebo. The main end-point measure used was the proportion of patients who achieved at least 30 percent reduction in pain from baseline to endpoint based on the Pain Visual Analog Scale (VAS). This outcome measure is viewed by both U.S. and EU regulatory authorities as a clinically meaningful endpoint. Results from the Fibromyalgia Impact Questionnaire (FIQ) were also considered - in the EU, the FIQ is considered equally relevant, whereas in the US, the FIQ is seen as supportive data.

In the top-line results, a significant number of patients treated with sodium oxybate achieved 30 percent or greater improvement in their pain. Of those patients receiving sodium oxybate treatment, 46.2% of patients on 4.5 g/night and 39.3% of patients on 6 g/night reported this level of pain relief, compared with only 27.3 percent of patients on placebo.

Patients' physical functioning and ability to perform daily tasks, as measured by the FIQ, were significantly different from placebo for the 4.5 g/night dose and approached significance for the 6 g/night dose.

The most common adverse events were were headache, nausea, dizziness, vomiting, diarrhea, anxiety, and sinusitis. Sodium oxybate was generally well tolerated, with the majority of adverse events reported being mild to moderate in nature.

Samuel Saks, M.D., Chief Executive Officer of Jazz Pharmaceuticals, said today that:

"Achieving positive sodium oxybate (JZP-6) data in fibromyalgia from this Phase III clinical trial is an exciting milestone".

Roch Doliveux, Chief Executive Officer of UCB, added:

"As UCB continues to focus on serious diseases of the central nervous system and immunology, we are delighted to partner with Jazz Pharmaceuticals in bringing new hope for patients with this under-treated condition."

Sodium oxybate is the active ingredient in Xyrem(R), a Jazz Pharmaceuticals product approved by the FDA for the treatment of excessive daytime sleepiness and cataplexy (the sudden loss of muscle tone) in patients with narcolepsy.

Only primary efficacy and safety data have been reviewed at this time and further analyses will be undertaken to examine the full results, including secondary endpoints, in greater detail.

The sodium oxybate Phase III clinical trial program also includes a second randomized, double blind, placebo-controlled study, which is continuing at sites in the U.S. and Europe. More than 90% of the subjects have been enrolled in this second Phase III trial.

Jazz Pharmaceuticals anticipates submitting a New Drug Application for sodium oxybate to the U.S. Food and Drug Administration by the end of 2009. UCB anticipates filing in the EU shortly after. UCB has the exclusive marketing and distribution rights to sodium oxybate for Fibromyalgia Syndrome in Europe and some other countries outside North America.