A new drug application (NDA) for a novel oxybate (JZP-258; Jazz Pharmaceuticals; Philadelphia, PA) has been submitted to the Food and Drug Administration (FDA). The submission is based on a phase 3 global double-blind placebo-controlled study that demonstrated the efficacy and safety of the oxybate in treating cataplexy and excessive daytime sleepiness (EDS) associated with narcolepsy.
The primary symptom of narcolepsy is EDS,which is present in all people with the disorder. Characterized by the inability to stay awake and alert during the day, EDS results in drowsiness and unplanned lapses into sleep.
The exact mechanism of action of the novel oxybate is not fully understood, but it is thought that the therapeutic effects of the therapy on sleep/wake symptoms are mediated through modulation of GABAB receptor during sleep.
The novel oxybate is an investigational therapy for cataplexy and EDS in participants age 7 and more with narcolepsy. This oxybate has a unique composition of cations resulting in 92%, or about 1,000 mg to 1,500 mg, less sodium than sodium oxybate (Xyrem; Jazz Pharmaceuticals) ). This substantial reduction in sodium could enable participants to achieve a daily sodium intake target of 2,300 mg, and ideally 1,500 mg, as set by the American Heart Association.
"Narcolepsy is a chronic disorder, for which lifelong therapy may be clinically indicated, and is associated with an increased risk of comorbid conditions, including hypertension and cardiovascular disease," said Robert Iannone, MD, MSCE, executive vice president, research and development, Jazz Pharmaceuticals.
Thomas P. Leist, MD
Mark B. Skeen, MD
Crystal Dixon, MD, Miguel Melo-Bicchi, MD, and Kottil W. Rammohan, MD