The National Organization for Rare Disorders (NORD) is pleased to announce that the FDA has approved Xyrem (sodium oxybate) oral solution to treat cataplexy, a rare debilitating disease associated with Narcolepsy. Xyrem is the first and only FDA approved treatment for this rare disorder.
“The approval of Xyrem is a triumph for sufferers of cataplexy and others with rare diseases,” said Abbey Meyers, President of NORD. “This milestone approval marks the commitment so many parties have made to ensure that an unmet medical need would be addressed appropriately. While we recognize this one victory, we will continue our diligence to bring national attention desperate people with unmet medical needs for whom promising therapeutics could bring normalcy back to their lives,” she continued.
Cataplexy is a disorder associated with Narcolepsy, a chronic neurological disorder that affects approximately 140,000 Americans. An estimated 60-90% of Narcolepsy patients experience the debilitating symptom of cataplexy, which is a sudden loss of muscular control and weakness triggered by emotions such as amusement, anger, sadness or surprise. The effects of cataplexy range from dropping of the jaw and slumping of the head, to buckling of the legs and even collapse of the whole body. These effects can last for a few seconds to several minutes. Xyrem, developed by Orphan Medical Inc. of Minnetonka, MN, will help to effectively reduce the symptoms of cataplexy by some 90%.
“Education is the most important next step in supporting the patient population by driving awareness of the disease,” said Ms. Meyers. “Xyrem provides a new treatment option for this very debilitating disorder and we commend Orphan Medical’s strategy to educate physicians to ensure that Xyrem is used responsibly and according to prescription.”
About the National Organization for Rare Disorders (NORD)
The National Organization for Rare Disorders (NORD) is a unique federation of voluntary health organizations dedicated to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service. To learn more about NORD’s role in improving the lives of over 25 million Americans affected by one of the 6,000 rare diseases, visit their website at www.rarediseases.org.
Check here to read about events on Capitol Hill, funding for rare-disease research, and other topics of interest from NORD's office in Washington, DC.