ORPHAN MEDICAL REPORTS ON FIBROMYALGIA SYNDROME SCIENTIFIC ADVISORY MEETING
September 4, 2003.
MINNEAPOLIS – September 4, 2003 – Orphan Medical (Nasdaq: ORPH) announced at the Thomas Weisel Healthcare Conference that it recently held a scientific advisory meeting with prominent clinical specialists in fibromyalgia syndrome (FMS). The meeting identified measurement endpoints for a proof-of-principle trial to assess Xyrem® (sodium oxybate) oral solution as a treatment for the symptoms of fibromyalgia. Orphan Medical expects to submit an Investigational New Drug (IND) application and protocol for this trial to the U.S. Food and Drug Administration (FDA) later this year.
“Our advisory meeting participants strongly supported evaluating Xyrem in the treatment of fibromyalgia based on early published data, current clinical experience, and its known pharmacology,” said William Houghton, M.D., Chief Scientific and Medical Officer for Orphan Medical. “Sleep disturbance and daytime fatigue are important components of fibromyalgia. The known impact of Xyrem on sleep architecture provides a rationale for this potential use. We are seeking to address an unmet medical need as there are currently no approved medications for the treatment of fibromyalgia which may affect up to two percent of the population.”
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Fibromyalgia Syndrome is characterized by widespread muscle pain and stiffness. Its incidence in women is three times that of men. Other symptoms include persistent fatigue, headaches, cognitive or memory impairment, morning stiffness and non-restorative sleep.
“Clinical acceptance of Xyrem as a treatment for cataplexy in narcolepsy is growing and the next step in our Xyrem life cycle management program is the evaluation of Xyrem in treating excessive daytime sleepiness associated with narcolepsy,” said John Bullion, Chief Executive Officer of Orphan Medical. “The treatment of fibromyalgia is the first potential indication outside of narcolepsy. We are very encouraged by the positive support from the advisory meeting.”
The Company also indicated that the protocol for a Phase IV study required by the FDA to assess the effects of Xyrem in sleep-disordered breathing has been submitted and approved. Two sites specializing in sleep-disordered breathing have been identified and are awaiting Institutional Review Board approval. The Company will begin patient enrollment following these approvals.
Xyrem is the first and only approved medication for the treatment of cataplexy, a debilitating symptom of narcolepsy and was commercially launched in October of 2002. Cataplexy, a sudden loss of muscle tone, is usually triggered by strong emotions such as laughter, anger, or surprise. In its most severe form, cataplexy can cause a person to collapse. Sixty to ninety percent of the 140,000 Americans with narcolepsy suffer from cataplexy.
Orphan Medical acquires, develops, and markets pharmaceuticals of high medical value for inadequately treated and uncommon diseases treated by specialist physicians. Orphan Medical intends to build a strong presence in the sleep and central nervous system (CNS) markets. Orphan Medical’s Internet Web site address is www.orphan.com